Translating the Genomics Revolution: The Need for an International Gene Therapy Consortium for Monogenic Diseases
Identifieur interne : 004425 ( Main/Exploration ); précédent : 004424; suivant : 004426Translating the Genomics Revolution: The Need for an International Gene Therapy Consortium for Monogenic Diseases
Auteurs : Jacques P. Tremblay [Canada] ; Xiao Xiao [États-Unis] ; Annemieke Aartsma-Rus [Pays-Bas] ; Carlos Barbas [États-Unis] ; Helen M. Blau [États-Unis] ; Adam J. Bogdanove [États-Unis] ; Kym Boycott [Canada] ; Serge Braun [France] ; Xandra O. Breakefield [États-Unis] ; Juan A. Bueren [Espagne] ; Michael Buschmann [Canada] ; Barry J. Byrne [États-Unis] ; Michele Calos [États-Unis] ; Toni Cathomen [Allemagne] ; Jeffrey Chamberlain [États-Unis] ; Marinee Chuah [Belgique] ; Kenneth Cornetta [États-Unis] ; Kay E. Davies [Royaume-Uni] ; J George Dickson [Royaume-Uni] ; Philippe Duchateau [France] ; Terence R. Flotte [États-Unis] ; Daniel Gaudet [Canada] ; Charles A. Gersbach [États-Unis] ; Renald Gilbert [Canada] ; Joseph Glorioso [États-Unis] ; Roland W. Herzog [États-Unis] ; Katherine A. High [États-Unis] ; Wenlin Huang [République populaire de Chine] ; Johnny Huard [États-Unis] ; J Keith Joung [États-Unis] ; Depei Liu [République populaire de Chine] ; Dexi Liu [États-Unis] ; Hanns Lochmüller [Royaume-Uni] ; Lawrence Lustig [États-Unis] ; Jeffrey Martens [États-Unis] ; Bernard Massie [Canada] ; Fulvio Mavilio [France] ; Jerry R. Mendell [États-Unis] ; Amit Nathwani [Royaume-Uni] ; Katherine Ponder [États-Unis] ; Matthew Porteus [États-Unis] ; Jack Puymirat [Canada] ; Jude Samulski [États-Unis] ; Shin'Ichi Takeda [Japon] ; Adrian Thrasher [Royaume-Uni] ; Thierry Vandendriessche [Belgique] ; Yuquan Wei [République populaire de Chine] ; James M. Wilson [États-Unis] ; Steve D. Wilton [Australie] ; John H. Wolfe [États-Unis] ; Guangping Gao [États-Unis]Source :
- Molecular Therapy [ 1525-0016 ] ; 2013.
Url:
DOI: 10.1038/mt.2013.4
PubMed: 23369965
PubMed Central: 3594019
Affiliations:
- Allemagne, Australie, Belgique, Canada, Espagne, France, Japon, Pays-Bas, Royaume-Uni, République populaire de Chine, États-Unis
Links toward previous steps (curation, corpus...)
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Le document en format XML
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Tremblay</name><affiliation wicri:level="1"><nlm:aff id="aff1"><institution>Centre de Recherche du CHU and Department of Molecular Medicine, Université Laval</institution>, Quebec, Quebec,<country>Canada</country></nlm:aff><country xml:lang="fr">Canada</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Xiao, Xiao" sort="Xiao, Xiao" uniqKey="Xiao X" first="Xiao" last="Xiao">Xiao Xiao</name><affiliation wicri:level="1"><nlm:aff id="aff2"><institution>University of North Carolina School of Pharmacy</institution>, Chapel Hill, North Carolina,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Aartsma Rus, Annemieke" sort="Aartsma Rus, Annemieke" uniqKey="Aartsma Rus A" first="Annemieke" last="Aartsma-Rus">Annemieke Aartsma-Rus</name><affiliation wicri:level="1"><nlm:aff id="aff3"><institution>Department of Human Genetics, Leiden University Medical Center</institution>, Leiden,<country>The Netherlands</country></nlm:aff><country xml:lang="fr">Pays-Bas</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Barbas, Carlos" sort="Barbas, Carlos" uniqKey="Barbas C" first="Carlos" last="Barbas">Carlos Barbas</name><affiliation wicri:level="1"><nlm:aff id="aff4"><institution>The Skaggs Institute for Chemical Biology and the Departments of Molecular Biology and Chemistry, The Scripps Research Institute</institution>, La Jolla, California,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Blau, Helen M" sort="Blau, Helen M" uniqKey="Blau H" first="Helen M" last="Blau">Helen M. Blau</name><affiliation wicri:level="1"><nlm:aff id="aff5"><institution>Baxter Laboratory for Stem Cell Biology, Stanford University School of Medicine</institution>, Stanford, California,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Bogdanove, Adam J" sort="Bogdanove, Adam J" uniqKey="Bogdanove A" first="Adam J" last="Bogdanove">Adam J. Bogdanove</name><affiliation wicri:level="1"><nlm:aff id="aff6"><institution>Department of Plant Pathology and Plant-Microbe Biology, Cornell University</institution>, Ithaca, New York,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Boycott, Kym" sort="Boycott, Kym" uniqKey="Boycott K" first="Kym" last="Boycott">Kym Boycott</name><affiliation wicri:level="1"><nlm:aff id="aff7"><institution>Children's Hospital of Eastern Ontario Research Institute, University of Ottawa</institution>, Ottawa, Ontario,<country>Canada</country></nlm:aff><country xml:lang="fr">Canada</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Braun, Serge" sort="Braun, Serge" uniqKey="Braun S" first="Serge" last="Braun">Serge Braun</name><affiliation wicri:level="1"><nlm:aff id="aff8"><institution>AFM Telethon</institution>, Paris,<country>France</country></nlm:aff><country xml:lang="fr">France</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Breakefield, Xandra O" sort="Breakefield, Xandra O" uniqKey="Breakefield X" first="Xandra O" last="Breakefield">Xandra O. Breakefield</name><affiliation wicri:level="1"><nlm:aff id="aff9"><institution>Department of Neurology, Massachusetts General Hospital and Neuroscience Program, Harvard Medical School</institution>, Boston, Massachusetts,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Bueren, Juan A" sort="Bueren, Juan A" uniqKey="Bueren J" first="Juan A" last="Bueren">Juan A. Bueren</name><affiliation wicri:level="1"><nlm:aff id="aff10"><institution>Hematopoietic Innovative Therapies Division, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas and Centro de Investigación en Red de Enfermedades Raras (CIEMAT/CIBERER)</institution>, Madrid,<country>Spain</country></nlm:aff><country xml:lang="fr">Espagne</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Buschmann, Michael" sort="Buschmann, Michael" uniqKey="Buschmann M" first="Michael" last="Buschmann">Michael Buschmann</name><affiliation wicri:level="1"><nlm:aff id="aff11"><institution>Department of Chemical Engineering and Institute of Biomedical Engineering, Ecole Polytechnique, Montreal</institution>, Quebec,<country>Canada</country></nlm:aff><country xml:lang="fr">Canada</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Byrne, Barry J" sort="Byrne, Barry J" uniqKey="Byrne B" first="Barry J" last="Byrne">Barry J. Byrne</name><affiliation wicri:level="1"><nlm:aff id="aff12"><institution>Department of Pediatrics, University of Florida</institution>, Gainesville, Florida,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Calos, Michele" sort="Calos, Michele" uniqKey="Calos M" first="Michele" last="Calos">Michele Calos</name><affiliation wicri:level="1"><nlm:aff id="aff13"><institution>Department of Genetics, Stanford University School of Medicine</institution>, Stanford, California,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Cathomen, Toni" sort="Cathomen, Toni" uniqKey="Cathomen T" first="Toni" last="Cathomen">Toni Cathomen</name><affiliation wicri:level="1"><nlm:aff id="aff14"><institution>Laboratory of Cell and Gene Therapy, Center for Chronic Immunodeficiency, University Medical Center Freiburg</institution>, Freiburg,<country>Germany</country></nlm:aff><country xml:lang="fr">Allemagne</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Chamberlain, Jeffrey" sort="Chamberlain, Jeffrey" uniqKey="Chamberlain J" first="Jeffrey" last="Chamberlain">Jeffrey Chamberlain</name><affiliation wicri:level="1"><nlm:aff id="aff15"><institution>Departments of Neurology, Medicine, and Biochemistry, University of Washington School of Medicine</institution>, Seattle, Washington,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Chuah, Marinee" sort="Chuah, Marinee" uniqKey="Chuah M" first="Marinee" last="Chuah">Marinee Chuah</name><affiliation wicri:level="1"><nlm:aff id="aff16"><institution>Department of Gene Therapy and Regenerative Medicine, Vrije Universiteit Brussel and Department of Cardiovascular Sciences, Center for Molecular and Vascular Biology, University of Leuven</institution>, Leuven,<country>Belgium</country></nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Cornetta, Kenneth" sort="Cornetta, Kenneth" uniqKey="Cornetta K" first="Kenneth" last="Cornetta">Kenneth Cornetta</name><affiliation wicri:level="1"><nlm:aff id="aff17"><institution>Department of Medical and Molecular Genetics, Indiana University School of Medicine</institution>, Indianapolis, Indiana,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Davies, Kay E" sort="Davies, Kay E" uniqKey="Davies K" first="Kay E" last="Davies">Kay E. Davies</name><affiliation wicri:level="1"><nlm:aff id="aff18"><institution>MRC Functional Genomics Unit, University of Oxford</institution>, Oxford,<country>UK</country></nlm:aff><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Dickson, J George" sort="Dickson, J George" uniqKey="Dickson J" first="J George" last="Dickson">J George Dickson</name><affiliation wicri:level="1"><nlm:aff id="aff19"><institution>School of Biological Sciences, Royal Holloway–University of London</institution>, Egham, Surrey,<country>UK</country></nlm:aff><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Duchateau, Philippe" sort="Duchateau, Philippe" uniqKey="Duchateau P" first="Philippe" last="Duchateau">Philippe Duchateau</name><affiliation wicri:level="1"><nlm:aff id="aff20"><institution>Cellectis</institution>, Paris,<country>France</country></nlm:aff><country xml:lang="fr">France</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Flotte, Terence R" sort="Flotte, Terence R" uniqKey="Flotte T" first="Terence R" last="Flotte">Terence R. Flotte</name><affiliation wicri:level="1"><nlm:aff id="aff21"><institution>Gene Therapy Center and Department of Pediatrics, University of Massachusetts Medical School</institution>, Worcester, Massachusetts,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Gaudet, Daniel" sort="Gaudet, Daniel" uniqKey="Gaudet D" first="Daniel" last="Gaudet">Daniel Gaudet</name><affiliation wicri:level="1"><nlm:aff id="aff22"><institution>Department of Medicine, Université de Montréal</institution>, Montreal, Quebec,<country>Canada</country></nlm:aff><country xml:lang="fr">Canada</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Gersbach, Charles A" sort="Gersbach, Charles A" uniqKey="Gersbach C" first="Charles A" last="Gersbach">Charles A. Gersbach</name><affiliation wicri:level="1"><nlm:aff id="aff23"><institution>Department of Biomedical Engineering and Institute for Genome Science and Policy, Duke University, Durham</institution>, North Carolina,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Gilbert, Renald" sort="Gilbert, Renald" uniqKey="Gilbert R" first="Renald" last="Gilbert">Renald Gilbert</name><affiliation wicri:level="1"><nlm:aff id="aff24"><institution>Biotechnology Research Institute</institution>, Montreal, Quebec,<country>Canada</country></nlm:aff><country xml:lang="fr">Canada</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Glorioso, Joseph" sort="Glorioso, Joseph" uniqKey="Glorioso J" first="Joseph" last="Glorioso">Joseph Glorioso</name><affiliation wicri:level="1"><nlm:aff id="aff25"><institution>Department of Microbiology and Molecular Genetics, School of Medicine, University of Pittsburgh</institution>, Pittsburgh, Pennsylvania,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Herzog, Roland W" sort="Herzog, Roland W" uniqKey="Herzog R" first="Roland W" last="Herzog">Roland W. Herzog</name><affiliation wicri:level="1"><nlm:aff id="aff26"><institution>Department of Pediatrics, University of Florida</institution>, Florida,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="High, Katherine A" sort="High, Katherine A" uniqKey="High K" first="Katherine A" last="High">Katherine A. High</name><affiliation wicri:level="1"><nlm:aff id="aff27"><institution>Perelman School of Medicine at the University of Pennsylvania and Howard Hughes Medical Institute at the Children's Hospital of Philadelphia</institution>, Philadelphia, Pennsylvania,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Huang, Wenlin" sort="Huang, Wenlin" uniqKey="Huang W" first="Wenlin" last="Huang">Wenlin Huang</name><affiliation wicri:level="1"><nlm:aff id="aff28"><institution>State Key Laboratory of Oncology in South China, Cancer Center, Sun Yat-Sen University</institution>, Guangzhou,<country>China</country></nlm:aff><country xml:lang="fr">République populaire de Chine</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Huard, Johnny" sort="Huard, Johnny" uniqKey="Huard J" first="Johnny" last="Huard">Johnny Huard</name><affiliation wicri:level="1"><nlm:aff id="aff29"><institution>Department of Orthopaedic Surgery, University of Pittsburgh</institution>, Pittsburgh, Pennsylvania,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Joung, J Keith" sort="Joung, J Keith" uniqKey="Joung J" first="J Keith" last="Joung">J Keith Joung</name><affiliation wicri:level="1"><nlm:aff id="aff30"><institution>Molecular Pathology Unit, Massachusetts General Hospital, Charlestown and Department of Pathology, Harvard Medical School</institution>, Boston, Massachusetts,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Liu, Depei" sort="Liu, Depei" uniqKey="Liu D" first="Depei" last="Liu">Depei Liu</name><affiliation wicri:level="1"><nlm:aff id="aff31"><institution>Department of Biochemistry and Molecular Biology, State Key Laboratory of Medical Molecular Biology, Institute of Basic Medical Sciences, Chinese Academy of Medical Sciences and Peking Union Medical College</institution>, Beijing,<country>China</country></nlm:aff><country xml:lang="fr">République populaire de Chine</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Liu, Dexi" sort="Liu, Dexi" uniqKey="Liu D" first="Dexi" last="Liu">Dexi Liu</name><affiliation wicri:level="1"><nlm:aff id="aff32"><institution>Department of Pharmaceutical and Biomedical Sciences, University of Georgia College of Pharmacy</institution>, Athens, Georgia,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Lochmuller, Hanns" sort="Lochmuller, Hanns" uniqKey="Lochmuller H" first="Hanns" last="Lochmüller">Hanns Lochmüller</name><affiliation wicri:level="1"><nlm:aff id="aff33"><institution>Institute of Genetic Medicine, Newcastle University</institution>, Newcastle Upon Tyne,<country>UK</country></nlm:aff><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Lustig, Lawrence" sort="Lustig, Lawrence" uniqKey="Lustig L" first="Lawrence" last="Lustig">Lawrence Lustig</name><affiliation wicri:level="1"><nlm:aff id="aff34"><institution>Department of Otolaryngology–Head and Neck Surgery, University of California, San Francisco</institution>, San Francisco, California,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Martens, Jeffrey" sort="Martens, Jeffrey" uniqKey="Martens J" first="Jeffrey" last="Martens">Jeffrey Martens</name><affiliation wicri:level="1"><nlm:aff id="aff35"><institution>Department of Pharmacology, University of Michigan Medical School</institution>, Ann Arbor, Michigan,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Massie, Bernard" sort="Massie, Bernard" uniqKey="Massie B" first="Bernard" last="Massie">Bernard Massie</name><affiliation wicri:level="1"><nlm:aff id="aff36"><institution>Biotechnology Research Institute</institution>, Montreal, Quebec,<country>Canada</country></nlm:aff><country xml:lang="fr">Canada</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Mavilio, Fulvio" sort="Mavilio, Fulvio" uniqKey="Mavilio F" first="Fulvio" last="Mavilio">Fulvio Mavilio</name><affiliation wicri:level="1"><nlm:aff id="aff37"><institution>Genethon</institution>, Evry,<country>France</country></nlm:aff><country xml:lang="fr">France</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Mendell, Jerry R" sort="Mendell, Jerry R" uniqKey="Mendell J" first="Jerry R" last="Mendell">Jerry R. Mendell</name><affiliation wicri:level="1"><nlm:aff id="aff38"><institution>Paul D. Wellstone Center, Nationwide Children's Hospital and The Ohio State University</institution>, Columbus, Ohio,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Nathwani, Amit" sort="Nathwani, Amit" uniqKey="Nathwani A" first="Amit" last="Nathwani">Amit Nathwani</name><affiliation wicri:level="1"><nlm:aff id="aff39"><institution>Department of Haematology, UCL Cancer Institute</institution>, London,<country>UK</country></nlm:aff><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Ponder, Katherine" sort="Ponder, Katherine" uniqKey="Ponder K" first="Katherine" last="Ponder">Katherine Ponder</name><affiliation wicri:level="1"><nlm:aff id="aff40"><institution>Department of Internal Medicine, Washington University, St Louis</institution>, Missouri,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Porteus, Matthew" sort="Porteus, Matthew" uniqKey="Porteus M" first="Matthew" last="Porteus">Matthew Porteus</name><affiliation wicri:level="1"><nlm:aff id="aff41"><institution>Department of Pediatrics, Stanford University School of Medicine, Stanford</institution>, California,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Puymirat, Jack" sort="Puymirat, Jack" uniqKey="Puymirat J" first="Jack" last="Puymirat">Jack Puymirat</name><affiliation wicri:level="1"><nlm:aff id="aff42"><institution>Centre de Recherche du CHU and Department of Molecular Medicine, Université Laval, Quebec</institution>, Quebec,<country>Canada</country></nlm:aff><country xml:lang="fr">Canada</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Samulski, Jude" sort="Samulski, Jude" uniqKey="Samulski J" first="Jude" last="Samulski">Jude Samulski</name><affiliation wicri:level="1"><nlm:aff id="aff43"><institution>Cardiothoracic Division, Department of Surgery, Duke University Medical Center</institution>, Durham, North Carolina,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Takeda, Shin Ichi" sort="Takeda, Shin Ichi" uniqKey="Takeda S" first="Shin'Ichi" last="Takeda">Shin'Ichi Takeda</name><affiliation wicri:level="1"><nlm:aff id="aff44"><institution>National Center of Neurology and Psychiatry</institution>, Tokyo,<country>Japan</country></nlm:aff><country xml:lang="fr">Japon</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Thrasher, Adrian" sort="Thrasher, Adrian" uniqKey="Thrasher A" first="Adrian" last="Thrasher">Adrian Thrasher</name><affiliation wicri:level="1"><nlm:aff id="aff45"><institution>UCL Institute of Child Health</institution>, London,<country>UK</country></nlm:aff><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Vandendriessche, Thierry" sort="Vandendriessche, Thierry" uniqKey="Vandendriessche T" first="Thierry" last="Vandendriessche">Thierry Vandendriessche</name><affiliation wicri:level="1"><nlm:aff id="aff46"><institution>Department of Gene Therapy and Regenerative Medicine, Vrije Universiteit Brussel and Department of Cardiovascular Sciences, Center for Molecular and Vascular Biology, University of Leuven</institution>, Leuven,<country>Belgium</country></nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Wei, Yuquan" sort="Wei, Yuquan" uniqKey="Wei Y" first="Yuquan" last="Wei">Yuquan Wei</name><affiliation wicri:level="1"><nlm:aff id="aff47"><institution>National Gene Therapy Program, National Key Laboratory of Biotherapy, and Sichuan University</institution>, Chengdu,<country>China</country></nlm:aff><country xml:lang="fr">République populaire de Chine</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Wilson, James M" sort="Wilson, James M" uniqKey="Wilson J" first="James M" last="Wilson">James M. Wilson</name><affiliation wicri:level="1"><nlm:aff id="aff48"><institution>Department of Pathology and Laboratory Medicine, University of Pennsylvania</institution>, Philadelphia, Pennsylvania,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Wilton, Steve D" sort="Wilton, Steve D" uniqKey="Wilton S" first="Steve D" last="Wilton">Steve D. Wilton</name><affiliation wicri:level="1"><nlm:aff id="aff49"><institution>Centre for Neuromuscular and Neurological Disorders, University of Western Australia</institution>, Crawley,<country>Australia</country></nlm:aff><country xml:lang="fr">Australie</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Wolfe, John H" sort="Wolfe, John H" uniqKey="Wolfe J" first="John H" last="Wolfe">John H. Wolfe</name><affiliation wicri:level="1"><nlm:aff id="aff50"><institution>University of Pennsylvania and Children's Hospital of Philadelphia</institution>, Philadelphia, Pennsylvania,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Gao, Guangping" sort="Gao, Guangping" uniqKey="Gao G" first="Guangping" last="Gao">Guangping Gao</name><affiliation wicri:level="1"><nlm:aff id="aff51"><institution>University of Massachusetts Medical School</institution>, Worcester, MA,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author></titleStmt><publicationStmt><idno type="wicri:source">PMC</idno><idno type="pmid">23369965</idno><idno type="pmc">3594019</idno><idno type="url">http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3594019</idno><idno type="RBID">PMC:3594019</idno><idno type="doi">10.1038/mt.2013.4</idno><date when="2013">2013</date><idno type="wicri:Area/Pmc/Corpus">000009</idno><idno type="wicri:explorRef" wicri:stream="Pmc" wicri:step="Corpus" wicri:corpus="PMC">000009</idno><idno type="wicri:Area/Pmc/Curation">000009</idno><idno type="wicri:explorRef" wicri:stream="Pmc" wicri:step="Curation">000009</idno><idno type="wicri:Area/Pmc/Checkpoint">001820</idno><idno type="wicri:explorRef" wicri:stream="Pmc" wicri:step="Checkpoint">001820</idno><idno type="wicri:Area/Ncbi/Merge">001119</idno><idno type="wicri:Area/Ncbi/Curation">001119</idno><idno type="wicri:Area/Ncbi/Checkpoint">001119</idno><idno type="wicri:doubleKey">1525-0016:2013:Tremblay J:translating:the:genomics</idno><idno type="wicri:Area/Main/Merge">004506</idno><idno type="wicri:Area/Main/Curation">004425</idno><idno type="wicri:Area/Main/Exploration">004425</idno></publicationStmt><sourceDesc><biblStruct><analytic><title xml:lang="en" level="a" type="main">Translating the Genomics Revolution: The Need for an International Gene Therapy Consortium for Monogenic Diseases</title><author><name sortKey="Tremblay, Jacques P" sort="Tremblay, Jacques P" uniqKey="Tremblay J" first="Jacques P" last="Tremblay">Jacques P. Tremblay</name><affiliation wicri:level="1"><nlm:aff id="aff1"><institution>Centre de Recherche du CHU and Department of Molecular Medicine, Université Laval</institution>, Quebec, Quebec,<country>Canada</country></nlm:aff><country xml:lang="fr">Canada</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Xiao, Xiao" sort="Xiao, Xiao" uniqKey="Xiao X" first="Xiao" last="Xiao">Xiao Xiao</name><affiliation wicri:level="1"><nlm:aff id="aff2"><institution>University of North Carolina School of Pharmacy</institution>, Chapel Hill, North Carolina,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Aartsma Rus, Annemieke" sort="Aartsma Rus, Annemieke" uniqKey="Aartsma Rus A" first="Annemieke" last="Aartsma-Rus">Annemieke Aartsma-Rus</name><affiliation wicri:level="1"><nlm:aff id="aff3"><institution>Department of Human Genetics, Leiden University Medical Center</institution>, Leiden,<country>The Netherlands</country></nlm:aff><country xml:lang="fr">Pays-Bas</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Barbas, Carlos" sort="Barbas, Carlos" uniqKey="Barbas C" first="Carlos" last="Barbas">Carlos Barbas</name><affiliation wicri:level="1"><nlm:aff id="aff4"><institution>The Skaggs Institute for Chemical Biology and the Departments of Molecular Biology and Chemistry, The Scripps Research Institute</institution>, La Jolla, California,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Blau, Helen M" sort="Blau, Helen M" uniqKey="Blau H" first="Helen M" last="Blau">Helen M. Blau</name><affiliation wicri:level="1"><nlm:aff id="aff5"><institution>Baxter Laboratory for Stem Cell Biology, Stanford University School of Medicine</institution>, Stanford, California,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Bogdanove, Adam J" sort="Bogdanove, Adam J" uniqKey="Bogdanove A" first="Adam J" last="Bogdanove">Adam J. Bogdanove</name><affiliation wicri:level="1"><nlm:aff id="aff6"><institution>Department of Plant Pathology and Plant-Microbe Biology, Cornell University</institution>, Ithaca, New York,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Boycott, Kym" sort="Boycott, Kym" uniqKey="Boycott K" first="Kym" last="Boycott">Kym Boycott</name><affiliation wicri:level="1"><nlm:aff id="aff7"><institution>Children's Hospital of Eastern Ontario Research Institute, University of Ottawa</institution>, Ottawa, Ontario,<country>Canada</country></nlm:aff><country xml:lang="fr">Canada</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Braun, Serge" sort="Braun, Serge" uniqKey="Braun S" first="Serge" last="Braun">Serge Braun</name><affiliation wicri:level="1"><nlm:aff id="aff8"><institution>AFM Telethon</institution>, Paris,<country>France</country></nlm:aff><country xml:lang="fr">France</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Breakefield, Xandra O" sort="Breakefield, Xandra O" uniqKey="Breakefield X" first="Xandra O" last="Breakefield">Xandra O. Breakefield</name><affiliation wicri:level="1"><nlm:aff id="aff9"><institution>Department of Neurology, Massachusetts General Hospital and Neuroscience Program, Harvard Medical School</institution>, Boston, Massachusetts,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Bueren, Juan A" sort="Bueren, Juan A" uniqKey="Bueren J" first="Juan A" last="Bueren">Juan A. Bueren</name><affiliation wicri:level="1"><nlm:aff id="aff10"><institution>Hematopoietic Innovative Therapies Division, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas and Centro de Investigación en Red de Enfermedades Raras (CIEMAT/CIBERER)</institution>, Madrid,<country>Spain</country></nlm:aff><country xml:lang="fr">Espagne</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Buschmann, Michael" sort="Buschmann, Michael" uniqKey="Buschmann M" first="Michael" last="Buschmann">Michael Buschmann</name><affiliation wicri:level="1"><nlm:aff id="aff11"><institution>Department of Chemical Engineering and Institute of Biomedical Engineering, Ecole Polytechnique, Montreal</institution>, Quebec,<country>Canada</country></nlm:aff><country xml:lang="fr">Canada</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Byrne, Barry J" sort="Byrne, Barry J" uniqKey="Byrne B" first="Barry J" last="Byrne">Barry J. Byrne</name><affiliation wicri:level="1"><nlm:aff id="aff12"><institution>Department of Pediatrics, University of Florida</institution>, Gainesville, Florida,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Calos, Michele" sort="Calos, Michele" uniqKey="Calos M" first="Michele" last="Calos">Michele Calos</name><affiliation wicri:level="1"><nlm:aff id="aff13"><institution>Department of Genetics, Stanford University School of Medicine</institution>, Stanford, California,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Cathomen, Toni" sort="Cathomen, Toni" uniqKey="Cathomen T" first="Toni" last="Cathomen">Toni Cathomen</name><affiliation wicri:level="1"><nlm:aff id="aff14"><institution>Laboratory of Cell and Gene Therapy, Center for Chronic Immunodeficiency, University Medical Center Freiburg</institution>, Freiburg,<country>Germany</country></nlm:aff><country xml:lang="fr">Allemagne</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Chamberlain, Jeffrey" sort="Chamberlain, Jeffrey" uniqKey="Chamberlain J" first="Jeffrey" last="Chamberlain">Jeffrey Chamberlain</name><affiliation wicri:level="1"><nlm:aff id="aff15"><institution>Departments of Neurology, Medicine, and Biochemistry, University of Washington School of Medicine</institution>, Seattle, Washington,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Chuah, Marinee" sort="Chuah, Marinee" uniqKey="Chuah M" first="Marinee" last="Chuah">Marinee Chuah</name><affiliation wicri:level="1"><nlm:aff id="aff16"><institution>Department of Gene Therapy and Regenerative Medicine, Vrije Universiteit Brussel and Department of Cardiovascular Sciences, Center for Molecular and Vascular Biology, University of Leuven</institution>, Leuven,<country>Belgium</country></nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Cornetta, Kenneth" sort="Cornetta, Kenneth" uniqKey="Cornetta K" first="Kenneth" last="Cornetta">Kenneth Cornetta</name><affiliation wicri:level="1"><nlm:aff id="aff17"><institution>Department of Medical and Molecular Genetics, Indiana University School of Medicine</institution>, Indianapolis, Indiana,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Davies, Kay E" sort="Davies, Kay E" uniqKey="Davies K" first="Kay E" last="Davies">Kay E. Davies</name><affiliation wicri:level="1"><nlm:aff id="aff18"><institution>MRC Functional Genomics Unit, University of Oxford</institution>, Oxford,<country>UK</country></nlm:aff><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Dickson, J George" sort="Dickson, J George" uniqKey="Dickson J" first="J George" last="Dickson">J George Dickson</name><affiliation wicri:level="1"><nlm:aff id="aff19"><institution>School of Biological Sciences, Royal Holloway–University of London</institution>, Egham, Surrey,<country>UK</country></nlm:aff><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Duchateau, Philippe" sort="Duchateau, Philippe" uniqKey="Duchateau P" first="Philippe" last="Duchateau">Philippe Duchateau</name><affiliation wicri:level="1"><nlm:aff id="aff20"><institution>Cellectis</institution>, Paris,<country>France</country></nlm:aff><country xml:lang="fr">France</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Flotte, Terence R" sort="Flotte, Terence R" uniqKey="Flotte T" first="Terence R" last="Flotte">Terence R. Flotte</name><affiliation wicri:level="1"><nlm:aff id="aff21"><institution>Gene Therapy Center and Department of Pediatrics, University of Massachusetts Medical School</institution>, Worcester, Massachusetts,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Gaudet, Daniel" sort="Gaudet, Daniel" uniqKey="Gaudet D" first="Daniel" last="Gaudet">Daniel Gaudet</name><affiliation wicri:level="1"><nlm:aff id="aff22"><institution>Department of Medicine, Université de Montréal</institution>, Montreal, Quebec,<country>Canada</country></nlm:aff><country xml:lang="fr">Canada</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Gersbach, Charles A" sort="Gersbach, Charles A" uniqKey="Gersbach C" first="Charles A" last="Gersbach">Charles A. Gersbach</name><affiliation wicri:level="1"><nlm:aff id="aff23"><institution>Department of Biomedical Engineering and Institute for Genome Science and Policy, Duke University, Durham</institution>, North Carolina,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Gilbert, Renald" sort="Gilbert, Renald" uniqKey="Gilbert R" first="Renald" last="Gilbert">Renald Gilbert</name><affiliation wicri:level="1"><nlm:aff id="aff24"><institution>Biotechnology Research Institute</institution>, Montreal, Quebec,<country>Canada</country></nlm:aff><country xml:lang="fr">Canada</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Glorioso, Joseph" sort="Glorioso, Joseph" uniqKey="Glorioso J" first="Joseph" last="Glorioso">Joseph Glorioso</name><affiliation wicri:level="1"><nlm:aff id="aff25"><institution>Department of Microbiology and Molecular Genetics, School of Medicine, University of Pittsburgh</institution>, Pittsburgh, Pennsylvania,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Herzog, Roland W" sort="Herzog, Roland W" uniqKey="Herzog R" first="Roland W" last="Herzog">Roland W. Herzog</name><affiliation wicri:level="1"><nlm:aff id="aff26"><institution>Department of Pediatrics, University of Florida</institution>, Florida,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="High, Katherine A" sort="High, Katherine A" uniqKey="High K" first="Katherine A" last="High">Katherine A. High</name><affiliation wicri:level="1"><nlm:aff id="aff27"><institution>Perelman School of Medicine at the University of Pennsylvania and Howard Hughes Medical Institute at the Children's Hospital of Philadelphia</institution>, Philadelphia, Pennsylvania,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Huang, Wenlin" sort="Huang, Wenlin" uniqKey="Huang W" first="Wenlin" last="Huang">Wenlin Huang</name><affiliation wicri:level="1"><nlm:aff id="aff28"><institution>State Key Laboratory of Oncology in South China, Cancer Center, Sun Yat-Sen University</institution>, Guangzhou,<country>China</country></nlm:aff><country xml:lang="fr">République populaire de Chine</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Huard, Johnny" sort="Huard, Johnny" uniqKey="Huard J" first="Johnny" last="Huard">Johnny Huard</name><affiliation wicri:level="1"><nlm:aff id="aff29"><institution>Department of Orthopaedic Surgery, University of Pittsburgh</institution>, Pittsburgh, Pennsylvania,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Joung, J Keith" sort="Joung, J Keith" uniqKey="Joung J" first="J Keith" last="Joung">J Keith Joung</name><affiliation wicri:level="1"><nlm:aff id="aff30"><institution>Molecular Pathology Unit, Massachusetts General Hospital, Charlestown and Department of Pathology, Harvard Medical School</institution>, Boston, Massachusetts,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Liu, Depei" sort="Liu, Depei" uniqKey="Liu D" first="Depei" last="Liu">Depei Liu</name><affiliation wicri:level="1"><nlm:aff id="aff31"><institution>Department of Biochemistry and Molecular Biology, State Key Laboratory of Medical Molecular Biology, Institute of Basic Medical Sciences, Chinese Academy of Medical Sciences and Peking Union Medical College</institution>, Beijing,<country>China</country></nlm:aff><country xml:lang="fr">République populaire de Chine</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Liu, Dexi" sort="Liu, Dexi" uniqKey="Liu D" first="Dexi" last="Liu">Dexi Liu</name><affiliation wicri:level="1"><nlm:aff id="aff32"><institution>Department of Pharmaceutical and Biomedical Sciences, University of Georgia College of Pharmacy</institution>, Athens, Georgia,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Lochmuller, Hanns" sort="Lochmuller, Hanns" uniqKey="Lochmuller H" first="Hanns" last="Lochmüller">Hanns Lochmüller</name><affiliation wicri:level="1"><nlm:aff id="aff33"><institution>Institute of Genetic Medicine, Newcastle University</institution>, Newcastle Upon Tyne,<country>UK</country></nlm:aff><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Lustig, Lawrence" sort="Lustig, Lawrence" uniqKey="Lustig L" first="Lawrence" last="Lustig">Lawrence Lustig</name><affiliation wicri:level="1"><nlm:aff id="aff34"><institution>Department of Otolaryngology–Head and Neck Surgery, University of California, San Francisco</institution>, San Francisco, California,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Martens, Jeffrey" sort="Martens, Jeffrey" uniqKey="Martens J" first="Jeffrey" last="Martens">Jeffrey Martens</name><affiliation wicri:level="1"><nlm:aff id="aff35"><institution>Department of Pharmacology, University of Michigan Medical School</institution>, Ann Arbor, Michigan,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Massie, Bernard" sort="Massie, Bernard" uniqKey="Massie B" first="Bernard" last="Massie">Bernard Massie</name><affiliation wicri:level="1"><nlm:aff id="aff36"><institution>Biotechnology Research Institute</institution>, Montreal, Quebec,<country>Canada</country></nlm:aff><country xml:lang="fr">Canada</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Mavilio, Fulvio" sort="Mavilio, Fulvio" uniqKey="Mavilio F" first="Fulvio" last="Mavilio">Fulvio Mavilio</name><affiliation wicri:level="1"><nlm:aff id="aff37"><institution>Genethon</institution>, Evry,<country>France</country></nlm:aff><country xml:lang="fr">France</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Mendell, Jerry R" sort="Mendell, Jerry R" uniqKey="Mendell J" first="Jerry R" last="Mendell">Jerry R. Mendell</name><affiliation wicri:level="1"><nlm:aff id="aff38"><institution>Paul D. Wellstone Center, Nationwide Children's Hospital and The Ohio State University</institution>, Columbus, Ohio,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Nathwani, Amit" sort="Nathwani, Amit" uniqKey="Nathwani A" first="Amit" last="Nathwani">Amit Nathwani</name><affiliation wicri:level="1"><nlm:aff id="aff39"><institution>Department of Haematology, UCL Cancer Institute</institution>, London,<country>UK</country></nlm:aff><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Ponder, Katherine" sort="Ponder, Katherine" uniqKey="Ponder K" first="Katherine" last="Ponder">Katherine Ponder</name><affiliation wicri:level="1"><nlm:aff id="aff40"><institution>Department of Internal Medicine, Washington University, St Louis</institution>, Missouri,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Porteus, Matthew" sort="Porteus, Matthew" uniqKey="Porteus M" first="Matthew" last="Porteus">Matthew Porteus</name><affiliation wicri:level="1"><nlm:aff id="aff41"><institution>Department of Pediatrics, Stanford University School of Medicine, Stanford</institution>, California,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Puymirat, Jack" sort="Puymirat, Jack" uniqKey="Puymirat J" first="Jack" last="Puymirat">Jack Puymirat</name><affiliation wicri:level="1"><nlm:aff id="aff42"><institution>Centre de Recherche du CHU and Department of Molecular Medicine, Université Laval, Quebec</institution>, Quebec,<country>Canada</country></nlm:aff><country xml:lang="fr">Canada</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Samulski, Jude" sort="Samulski, Jude" uniqKey="Samulski J" first="Jude" last="Samulski">Jude Samulski</name><affiliation wicri:level="1"><nlm:aff id="aff43"><institution>Cardiothoracic Division, Department of Surgery, Duke University Medical Center</institution>, Durham, North Carolina,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Takeda, Shin Ichi" sort="Takeda, Shin Ichi" uniqKey="Takeda S" first="Shin'Ichi" last="Takeda">Shin'Ichi Takeda</name><affiliation wicri:level="1"><nlm:aff id="aff44"><institution>National Center of Neurology and Psychiatry</institution>, Tokyo,<country>Japan</country></nlm:aff><country xml:lang="fr">Japon</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Thrasher, Adrian" sort="Thrasher, Adrian" uniqKey="Thrasher A" first="Adrian" last="Thrasher">Adrian Thrasher</name><affiliation wicri:level="1"><nlm:aff id="aff45"><institution>UCL Institute of Child Health</institution>, London,<country>UK</country></nlm:aff><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Vandendriessche, Thierry" sort="Vandendriessche, Thierry" uniqKey="Vandendriessche T" first="Thierry" last="Vandendriessche">Thierry Vandendriessche</name><affiliation wicri:level="1"><nlm:aff id="aff46"><institution>Department of Gene Therapy and Regenerative Medicine, Vrije Universiteit Brussel and Department of Cardiovascular Sciences, Center for Molecular and Vascular Biology, University of Leuven</institution>, Leuven,<country>Belgium</country></nlm:aff><country xml:lang="fr">Belgique</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Wei, Yuquan" sort="Wei, Yuquan" uniqKey="Wei Y" first="Yuquan" last="Wei">Yuquan Wei</name><affiliation wicri:level="1"><nlm:aff id="aff47"><institution>National Gene Therapy Program, National Key Laboratory of Biotherapy, and Sichuan University</institution>, Chengdu,<country>China</country></nlm:aff><country xml:lang="fr">République populaire de Chine</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Wilson, James M" sort="Wilson, James M" uniqKey="Wilson J" first="James M" last="Wilson">James M. Wilson</name><affiliation wicri:level="1"><nlm:aff id="aff48"><institution>Department of Pathology and Laboratory Medicine, University of Pennsylvania</institution>, Philadelphia, Pennsylvania,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Wilton, Steve D" sort="Wilton, Steve D" uniqKey="Wilton S" first="Steve D" last="Wilton">Steve D. Wilton</name><affiliation wicri:level="1"><nlm:aff id="aff49"><institution>Centre for Neuromuscular and Neurological Disorders, University of Western Australia</institution>, Crawley,<country>Australia</country></nlm:aff><country xml:lang="fr">Australie</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Wolfe, John H" sort="Wolfe, John H" uniqKey="Wolfe J" first="John H" last="Wolfe">John H. Wolfe</name><affiliation wicri:level="1"><nlm:aff id="aff50"><institution>University of Pennsylvania and Children's Hospital of Philadelphia</institution>, Philadelphia, Pennsylvania,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author><author><name sortKey="Gao, Guangping" sort="Gao, Guangping" uniqKey="Gao G" first="Guangping" last="Gao">Guangping Gao</name><affiliation wicri:level="1"><nlm:aff id="aff51"><institution>University of Massachusetts Medical School</institution>, Worcester, MA,<country>USA</country></nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea># see nlm:aff country strict</wicri:regionArea></affiliation></author></analytic><series><title level="j">Molecular Therapy</title><idno type="ISSN">1525-0016</idno><idno type="eISSN">1525-0024</idno><imprint><date when="2013">2013</date></imprint></series></biblStruct></sourceDesc></fileDesc><profileDesc><textClass></textClass></profileDesc></teiHeader><back><div1 type="bibliography"><listBibl><biblStruct><analytic><author><name sortKey="Takahashi, K" uniqKey="Takahashi K">K Takahashi</name></author><author><name sortKey="Yamanaka, S" uniqKey="Yamanaka S">S Yamanaka</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Gurdon, Jb" uniqKey="Gurdon J">JB Gurdon</name></author><author><name sortKey="Byrne, Ja" uniqKey="Byrne J">JA Byrne</name></author><author><name sortKey="Simonsson, S" uniqKey="Simonsson S">S Simonsson</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Gaudet, D" uniqKey="Gaudet D">D Gaudet</name></author><author><name sortKey="Methot, J" uniqKey="Methot J">J Méthot</name></author><author><name sortKey="Dery, S" uniqKey="Dery S">S Déry</name></author><author><name sortKey="Brisson, D" uniqKey="Brisson D">D Brisson</name></author><author><name sortKey="Essiembre, C" uniqKey="Essiembre C">C Essiembre</name></author><author><name sortKey="Tremblay, G" uniqKey="Tremblay G">G Tremblay</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Mavilio, F" uniqKey="Mavilio F">F Mavilio</name></author></analytic></biblStruct></listBibl></div1></back></TEI><affiliations><list><country><li>Allemagne</li><li>Australie</li><li>Belgique</li><li>Canada</li><li>Espagne</li><li>France</li><li>Japon</li><li>Pays-Bas</li><li>Royaume-Uni</li><li>République populaire de Chine</li><li>États-Unis</li></country></list><tree><country name="Canada"><noRegion><name sortKey="Tremblay, Jacques P" sort="Tremblay, Jacques P" uniqKey="Tremblay J" first="Jacques P" last="Tremblay">Jacques P. Tremblay</name></noRegion><name sortKey="Boycott, Kym" sort="Boycott, Kym" uniqKey="Boycott K" first="Kym" last="Boycott">Kym Boycott</name><name sortKey="Buschmann, Michael" sort="Buschmann, Michael" uniqKey="Buschmann M" first="Michael" last="Buschmann">Michael Buschmann</name><name sortKey="Gaudet, Daniel" sort="Gaudet, Daniel" uniqKey="Gaudet D" first="Daniel" last="Gaudet">Daniel Gaudet</name><name sortKey="Gilbert, Renald" sort="Gilbert, Renald" uniqKey="Gilbert R" first="Renald" last="Gilbert">Renald Gilbert</name><name sortKey="Massie, Bernard" sort="Massie, Bernard" uniqKey="Massie B" first="Bernard" last="Massie">Bernard Massie</name><name sortKey="Puymirat, Jack" sort="Puymirat, Jack" uniqKey="Puymirat J" first="Jack" last="Puymirat">Jack Puymirat</name></country><country name="États-Unis"><noRegion><name sortKey="Xiao, Xiao" sort="Xiao, Xiao" uniqKey="Xiao X" first="Xiao" last="Xiao">Xiao Xiao</name></noRegion><name sortKey="Barbas, Carlos" sort="Barbas, Carlos" uniqKey="Barbas C" first="Carlos" last="Barbas">Carlos Barbas</name><name sortKey="Blau, Helen M" sort="Blau, Helen M" uniqKey="Blau H" first="Helen M" last="Blau">Helen M. Blau</name><name sortKey="Bogdanove, Adam J" sort="Bogdanove, Adam J" uniqKey="Bogdanove A" first="Adam J" last="Bogdanove">Adam J. Bogdanove</name><name sortKey="Breakefield, Xandra O" sort="Breakefield, Xandra O" uniqKey="Breakefield X" first="Xandra O" last="Breakefield">Xandra O. Breakefield</name><name sortKey="Byrne, Barry J" sort="Byrne, Barry J" uniqKey="Byrne B" first="Barry J" last="Byrne">Barry J. Byrne</name><name sortKey="Calos, Michele" sort="Calos, Michele" uniqKey="Calos M" first="Michele" last="Calos">Michele Calos</name><name sortKey="Chamberlain, Jeffrey" sort="Chamberlain, Jeffrey" uniqKey="Chamberlain J" first="Jeffrey" last="Chamberlain">Jeffrey Chamberlain</name><name sortKey="Cornetta, Kenneth" sort="Cornetta, Kenneth" uniqKey="Cornetta K" first="Kenneth" last="Cornetta">Kenneth Cornetta</name><name sortKey="Flotte, Terence R" sort="Flotte, Terence R" uniqKey="Flotte T" first="Terence R" last="Flotte">Terence R. Flotte</name><name sortKey="Gao, Guangping" sort="Gao, Guangping" uniqKey="Gao G" first="Guangping" last="Gao">Guangping Gao</name><name sortKey="Gersbach, Charles A" sort="Gersbach, Charles A" uniqKey="Gersbach C" first="Charles A" last="Gersbach">Charles A. Gersbach</name><name sortKey="Glorioso, Joseph" sort="Glorioso, Joseph" uniqKey="Glorioso J" first="Joseph" last="Glorioso">Joseph Glorioso</name><name sortKey="Herzog, Roland W" sort="Herzog, Roland W" uniqKey="Herzog R" first="Roland W" last="Herzog">Roland W. Herzog</name><name sortKey="High, Katherine A" sort="High, Katherine A" uniqKey="High K" first="Katherine A" last="High">Katherine A. High</name><name sortKey="Huard, Johnny" sort="Huard, Johnny" uniqKey="Huard J" first="Johnny" last="Huard">Johnny Huard</name><name sortKey="Joung, J Keith" sort="Joung, J Keith" uniqKey="Joung J" first="J Keith" last="Joung">J Keith Joung</name><name sortKey="Liu, Dexi" sort="Liu, Dexi" uniqKey="Liu D" first="Dexi" last="Liu">Dexi Liu</name><name sortKey="Lustig, Lawrence" sort="Lustig, Lawrence" uniqKey="Lustig L" first="Lawrence" last="Lustig">Lawrence Lustig</name><name sortKey="Martens, Jeffrey" sort="Martens, Jeffrey" uniqKey="Martens J" first="Jeffrey" last="Martens">Jeffrey Martens</name><name sortKey="Mendell, Jerry R" sort="Mendell, Jerry R" uniqKey="Mendell J" first="Jerry R" last="Mendell">Jerry R. Mendell</name><name sortKey="Ponder, Katherine" sort="Ponder, Katherine" uniqKey="Ponder K" first="Katherine" last="Ponder">Katherine Ponder</name><name sortKey="Porteus, Matthew" sort="Porteus, Matthew" uniqKey="Porteus M" first="Matthew" last="Porteus">Matthew Porteus</name><name sortKey="Samulski, Jude" sort="Samulski, Jude" uniqKey="Samulski J" first="Jude" last="Samulski">Jude Samulski</name><name sortKey="Wilson, James M" sort="Wilson, James M" uniqKey="Wilson J" first="James M" last="Wilson">James M. Wilson</name><name sortKey="Wolfe, John H" sort="Wolfe, John H" uniqKey="Wolfe J" first="John H" last="Wolfe">John H. Wolfe</name></country><country name="Pays-Bas"><noRegion><name sortKey="Aartsma Rus, Annemieke" sort="Aartsma Rus, Annemieke" uniqKey="Aartsma Rus A" first="Annemieke" last="Aartsma-Rus">Annemieke Aartsma-Rus</name></noRegion></country><country name="France"><noRegion><name sortKey="Braun, Serge" sort="Braun, Serge" uniqKey="Braun S" first="Serge" last="Braun">Serge Braun</name></noRegion><name sortKey="Duchateau, Philippe" sort="Duchateau, Philippe" uniqKey="Duchateau P" first="Philippe" last="Duchateau">Philippe Duchateau</name><name sortKey="Mavilio, Fulvio" sort="Mavilio, Fulvio" uniqKey="Mavilio F" first="Fulvio" last="Mavilio">Fulvio Mavilio</name></country><country name="Espagne"><noRegion><name sortKey="Bueren, Juan A" sort="Bueren, Juan A" uniqKey="Bueren J" first="Juan A" last="Bueren">Juan A. Bueren</name></noRegion></country><country name="Allemagne"><noRegion><name sortKey="Cathomen, Toni" sort="Cathomen, Toni" uniqKey="Cathomen T" first="Toni" last="Cathomen">Toni Cathomen</name></noRegion></country><country name="Belgique"><noRegion><name sortKey="Chuah, Marinee" sort="Chuah, Marinee" uniqKey="Chuah M" first="Marinee" last="Chuah">Marinee Chuah</name></noRegion><name sortKey="Vandendriessche, Thierry" sort="Vandendriessche, Thierry" uniqKey="Vandendriessche T" first="Thierry" last="Vandendriessche">Thierry Vandendriessche</name></country><country name="Royaume-Uni"><noRegion><name sortKey="Davies, Kay E" sort="Davies, Kay E" uniqKey="Davies K" first="Kay E" last="Davies">Kay E. Davies</name></noRegion><name sortKey="Dickson, J George" sort="Dickson, J George" uniqKey="Dickson J" first="J George" last="Dickson">J George Dickson</name><name sortKey="Lochmuller, Hanns" sort="Lochmuller, Hanns" uniqKey="Lochmuller H" first="Hanns" last="Lochmüller">Hanns Lochmüller</name><name sortKey="Nathwani, Amit" sort="Nathwani, Amit" uniqKey="Nathwani A" first="Amit" last="Nathwani">Amit Nathwani</name><name sortKey="Thrasher, Adrian" sort="Thrasher, Adrian" uniqKey="Thrasher A" first="Adrian" last="Thrasher">Adrian Thrasher</name></country><country name="République populaire de Chine"><noRegion><name sortKey="Huang, Wenlin" sort="Huang, Wenlin" uniqKey="Huang W" first="Wenlin" last="Huang">Wenlin Huang</name></noRegion><name sortKey="Liu, Depei" sort="Liu, Depei" uniqKey="Liu D" first="Depei" last="Liu">Depei Liu</name><name sortKey="Wei, Yuquan" sort="Wei, Yuquan" uniqKey="Wei Y" first="Yuquan" last="Wei">Yuquan Wei</name></country><country name="Japon"><noRegion><name sortKey="Takeda, Shin Ichi" sort="Takeda, Shin Ichi" uniqKey="Takeda S" first="Shin'Ichi" last="Takeda">Shin'Ichi Takeda</name></noRegion></country><country name="Australie"><noRegion><name sortKey="Wilton, Steve D" sort="Wilton, Steve D" uniqKey="Wilton S" first="Steve D" last="Wilton">Steve D. Wilton</name></noRegion></country></tree></affiliations></record>Pour manipuler ce document sous Unix (Dilib)
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