Links to Exploration step
Le document en format XML
<record><TEI><teiHeader><fileDesc><titleStmt><title xml:lang="en">Safe, Efficient, and Reproducible Gene Therapy of the Brain in the Dog Models of Sanfilippo and Hurler Syndromes</title>
<author><name sortKey="Ellinwood, N Matthew" sort="Ellinwood, N Matthew" uniqKey="Ellinwood N" first="N Matthew" last="Ellinwood">N Matthew Ellinwood</name>
<affiliation><nlm:aff id="aff1"><institution>Department of Animal Science and the Center for Integrated Animal Genomics, Iowa State University</institution>
, Ames, Iowa,<country>USA</country>
</nlm:aff>
</affiliation>
<affiliation><nlm:aff id="aff2"><institution>Department of Veterinary Clinical Science, Iowa State University</institution>
, Ames, Iowa,<country>USA</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Ausseil, Jerome" sort="Ausseil, Jerome" uniqKey="Ausseil J" first="Jérôme" last="Ausseil">Jérôme Ausseil</name>
<affiliation><nlm:aff id="aff3"><institution>Institut Pasteur, Unité Rétrovirus et Transfert Génétique, Département de Neuroscience</institution>
, Paris,<country>France</country>
</nlm:aff>
</affiliation>
<affiliation><nlm:aff id="aff4"><institution>INSERM U622</institution>
, Paris,<country>France</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Desmaris, Nathalie" sort="Desmaris, Nathalie" uniqKey="Desmaris N" first="Nathalie" last="Desmaris">Nathalie Desmaris</name>
<affiliation><nlm:aff id="aff3"><institution>Institut Pasteur, Unité Rétrovirus et Transfert Génétique, Département de Neuroscience</institution>
, Paris,<country>France</country>
</nlm:aff>
</affiliation>
<affiliation><nlm:aff id="aff4"><institution>INSERM U622</institution>
, Paris,<country>France</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Bigou, Stephanie" sort="Bigou, Stephanie" uniqKey="Bigou S" first="Stéphanie" last="Bigou">Stéphanie Bigou</name>
<affiliation><nlm:aff id="aff3"><institution>Institut Pasteur, Unité Rétrovirus et Transfert Génétique, Département de Neuroscience</institution>
, Paris,<country>France</country>
</nlm:aff>
</affiliation>
<affiliation><nlm:aff id="aff4"><institution>INSERM U622</institution>
, Paris,<country>France</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Liu, Song" sort="Liu, Song" uniqKey="Liu S" first="Song" last="Liu">Song Liu</name>
<affiliation><nlm:aff id="aff3"><institution>Institut Pasteur, Unité Rétrovirus et Transfert Génétique, Département de Neuroscience</institution>
, Paris,<country>France</country>
</nlm:aff>
</affiliation>
<affiliation><nlm:aff id="aff4"><institution>INSERM U622</institution>
, Paris,<country>France</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Jens, Jackie K" sort="Jens, Jackie K" uniqKey="Jens J" first="Jackie K" last="Jens">Jackie K. Jens</name>
<affiliation><nlm:aff id="aff1"><institution>Department of Animal Science and the Center for Integrated Animal Genomics, Iowa State University</institution>
, Ames, Iowa,<country>USA</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Snella, Elizabeth M" sort="Snella, Elizabeth M" uniqKey="Snella E" first="Elizabeth M" last="Snella">Elizabeth M. Snella</name>
<affiliation><nlm:aff id="aff1"><institution>Department of Animal Science and the Center for Integrated Animal Genomics, Iowa State University</institution>
, Ames, Iowa,<country>USA</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Mohammed, Eman Ea" sort="Mohammed, Eman Ea" uniqKey="Mohammed E" first="Eman Ea" last="Mohammed">Eman Ea Mohammed</name>
<affiliation><nlm:aff id="aff1"><institution>Department of Animal Science and the Center for Integrated Animal Genomics, Iowa State University</institution>
, Ames, Iowa,<country>USA</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Thomson, Christopher B" sort="Thomson, Christopher B" uniqKey="Thomson C" first="Christopher B" last="Thomson">Christopher B. Thomson</name>
<affiliation><nlm:aff id="aff1"><institution>Department of Animal Science and the Center for Integrated Animal Genomics, Iowa State University</institution>
, Ames, Iowa,<country>USA</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Raoul, Sylvie" sort="Raoul, Sylvie" uniqKey="Raoul S" first="Sylvie" last="Raoul">Sylvie Raoul</name>
<affiliation><nlm:aff id="aff5"><institution>Service de Neurochirurgie, CHU Nord</institution>
, Nantes,<country>France</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Joussemet, Beatrice" sort="Joussemet, Beatrice" uniqKey="Joussemet B" first="Béatrice" last="Joussemet">Béatrice Joussemet</name>
<affiliation><nlm:aff id="aff6"><institution>Centre de Boisbonne, Ecole Nationale Vétérinaire, Agroalimentaire et de l'Alimentation Nantes-Atlantiques (Oniris)</institution>
, Nantes,<country>France</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Roux, Francoise" sort="Roux, Francoise" uniqKey="Roux F" first="Françoise" last="Roux">Françoise Roux</name>
<affiliation><nlm:aff id="aff6"><institution>Centre de Boisbonne, Ecole Nationale Vétérinaire, Agroalimentaire et de l'Alimentation Nantes-Atlantiques (Oniris)</institution>
, Nantes,<country>France</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Cherel, Yan" sort="Cherel, Yan" uniqKey="Cherel Y" first="Yan" last="Chérel">Yan Chérel</name>
<affiliation><nlm:aff id="aff7"><institution>UMR INRA 703, Ecole Nationale Vétérinaire, Agroalimentaire et de l'Alimentation Nantes-Atlantiques (Oniris)</institution>
, Nantes,<country>France</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Lajat, Yaouen" sort="Lajat, Yaouen" uniqKey="Lajat Y" first="Yaouen" last="Lajat">Yaouen Lajat</name>
<affiliation><nlm:aff id="aff5"><institution>Service de Neurochirurgie, CHU Nord</institution>
, Nantes,<country>France</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Piraud, Monique" sort="Piraud, Monique" uniqKey="Piraud M" first="Monique" last="Piraud">Monique Piraud</name>
<affiliation><nlm:aff id="aff8"><institution>Maladies Héréditaire du Métabolisme et Dépistage Néonatal, Centre de Biologie et de Pathologie, Groupement Hospitalier Est, CBPE</institution>
, Bron,<country>France</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Benchaouir, Rachid" sort="Benchaouir, Rachid" uniqKey="Benchaouir R" first="Rachid" last="Benchaouir">Rachid Benchaouir</name>
<affiliation><nlm:aff id="aff9"><institution>Amsterdam Molecular Therapeutics (AMT)</institution>
, Amsterdam,<country>The Netherlands</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Hermening, Stephan" sort="Hermening, Stephan" uniqKey="Hermening S" first="Stephan" last="Hermening">Stephan Hermening</name>
<affiliation><nlm:aff id="aff9"><institution>Amsterdam Molecular Therapeutics (AMT)</institution>
, Amsterdam,<country>The Netherlands</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Petry, Harald" sort="Petry, Harald" uniqKey="Petry H" first="Harald" last="Petry">Harald Petry</name>
<affiliation><nlm:aff id="aff9"><institution>Amsterdam Molecular Therapeutics (AMT)</institution>
, Amsterdam,<country>The Netherlands</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Froissart, Roseline" sort="Froissart, Roseline" uniqKey="Froissart R" first="Roseline" last="Froissart">Roseline Froissart</name>
<affiliation><nlm:aff id="aff8"><institution>Maladies Héréditaire du Métabolisme et Dépistage Néonatal, Centre de Biologie et de Pathologie, Groupement Hospitalier Est, CBPE</institution>
, Bron,<country>France</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Tardieu, Marc" sort="Tardieu, Marc" uniqKey="Tardieu M" first="Marc" last="Tardieu">Marc Tardieu</name>
<affiliation><nlm:aff id="aff10"><institution>Service de Neurologie Pédiatrique, Hôpital Bicêtre, Assistance Publique/Hôpitaux de Paris, INSERM U109</institution>
, le Kremlin-Bicêtre,<country>France</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Ciron, Carine" sort="Ciron, Carine" uniqKey="Ciron C" first="Carine" last="Ciron">Carine Ciron</name>
<affiliation><nlm:aff id="aff11"><institution>INSERM U649, Université de Nantes</institution>
, Nantes,<country>France</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Moullier, Philippe" sort="Moullier, Philippe" uniqKey="Moullier P" first="Philippe" last="Moullier">Philippe Moullier</name>
<affiliation><nlm:aff id="aff11"><institution>INSERM U649, Université de Nantes</institution>
, Nantes,<country>France</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Parkes, Jennifer" sort="Parkes, Jennifer" uniqKey="Parkes J" first="Jennifer" last="Parkes">Jennifer Parkes</name>
<affiliation><nlm:aff id="aff2"><institution>Department of Veterinary Clinical Science, Iowa State University</institution>
, Ames, Iowa,<country>USA</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Kline, Karen L" sort="Kline, Karen L" uniqKey="Kline K" first="Karen L" last="Kline">Karen L. Kline</name>
<affiliation><nlm:aff id="aff2"><institution>Department of Veterinary Clinical Science, Iowa State University</institution>
, Ames, Iowa,<country>USA</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Maire, Irene" sort="Maire, Irene" uniqKey="Maire I" first="Irène" last="Maire">Irène Maire</name>
<affiliation><nlm:aff id="aff8"><institution>Maladies Héréditaire du Métabolisme et Dépistage Néonatal, Centre de Biologie et de Pathologie, Groupement Hospitalier Est, CBPE</institution>
, Bron,<country>France</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Vanier, Marie Therese" sort="Vanier, Marie Therese" uniqKey="Vanier M" first="Marie-Thérèse" last="Vanier">Marie-Thérèse Vanier</name>
<affiliation><nlm:aff id="aff12"><institution>INSERM U820, Faculté de Médecine Laënnec</institution>
, Lyon,<country>France</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Heard, Jean Michel" sort="Heard, Jean Michel" uniqKey="Heard J" first="Jean-Michel" last="Heard">Jean-Michel Heard</name>
<affiliation><nlm:aff id="aff3"><institution>Institut Pasteur, Unité Rétrovirus et Transfert Génétique, Département de Neuroscience</institution>
, Paris,<country>France</country>
</nlm:aff>
</affiliation>
<affiliation><nlm:aff id="aff4"><institution>INSERM U622</institution>
, Paris,<country>France</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Colle, Marie Anne" sort="Colle, Marie Anne" uniqKey="Colle M" first="Marie-Anne" last="Colle">Marie-Anne Colle</name>
<affiliation><nlm:aff id="aff7"><institution>UMR INRA 703, Ecole Nationale Vétérinaire, Agroalimentaire et de l'Alimentation Nantes-Atlantiques (Oniris)</institution>
, Nantes,<country>France</country>
</nlm:aff>
</affiliation>
</author>
</titleStmt>
<publicationStmt><idno type="wicri:source">PMC</idno>
<idno type="pmid">21139569</idno>
<idno type="pmc">3034858</idno>
<idno type="url">http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3034858</idno>
<idno type="RBID">PMC:3034858</idno>
<idno type="doi">10.1038/mt.2010.265</idno>
<date when="2010">2010</date>
<idno type="wicri:Area/Pmc/Corpus">000007</idno>
<idno type="wicri:explorRef" wicri:stream="Pmc" wicri:step="Corpus" wicri:corpus="PMC">000007</idno>
</publicationStmt>
<sourceDesc><biblStruct><analytic><title xml:lang="en" level="a" type="main">Safe, Efficient, and Reproducible Gene Therapy of the Brain in the Dog Models of Sanfilippo and Hurler Syndromes</title>
<author><name sortKey="Ellinwood, N Matthew" sort="Ellinwood, N Matthew" uniqKey="Ellinwood N" first="N Matthew" last="Ellinwood">N Matthew Ellinwood</name>
<affiliation><nlm:aff id="aff1"><institution>Department of Animal Science and the Center for Integrated Animal Genomics, Iowa State University</institution>
, Ames, Iowa,<country>USA</country>
</nlm:aff>
</affiliation>
<affiliation><nlm:aff id="aff2"><institution>Department of Veterinary Clinical Science, Iowa State University</institution>
, Ames, Iowa,<country>USA</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Ausseil, Jerome" sort="Ausseil, Jerome" uniqKey="Ausseil J" first="Jérôme" last="Ausseil">Jérôme Ausseil</name>
<affiliation><nlm:aff id="aff3"><institution>Institut Pasteur, Unité Rétrovirus et Transfert Génétique, Département de Neuroscience</institution>
, Paris,<country>France</country>
</nlm:aff>
</affiliation>
<affiliation><nlm:aff id="aff4"><institution>INSERM U622</institution>
, Paris,<country>France</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Desmaris, Nathalie" sort="Desmaris, Nathalie" uniqKey="Desmaris N" first="Nathalie" last="Desmaris">Nathalie Desmaris</name>
<affiliation><nlm:aff id="aff3"><institution>Institut Pasteur, Unité Rétrovirus et Transfert Génétique, Département de Neuroscience</institution>
, Paris,<country>France</country>
</nlm:aff>
</affiliation>
<affiliation><nlm:aff id="aff4"><institution>INSERM U622</institution>
, Paris,<country>France</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Bigou, Stephanie" sort="Bigou, Stephanie" uniqKey="Bigou S" first="Stéphanie" last="Bigou">Stéphanie Bigou</name>
<affiliation><nlm:aff id="aff3"><institution>Institut Pasteur, Unité Rétrovirus et Transfert Génétique, Département de Neuroscience</institution>
, Paris,<country>France</country>
</nlm:aff>
</affiliation>
<affiliation><nlm:aff id="aff4"><institution>INSERM U622</institution>
, Paris,<country>France</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Liu, Song" sort="Liu, Song" uniqKey="Liu S" first="Song" last="Liu">Song Liu</name>
<affiliation><nlm:aff id="aff3"><institution>Institut Pasteur, Unité Rétrovirus et Transfert Génétique, Département de Neuroscience</institution>
, Paris,<country>France</country>
</nlm:aff>
</affiliation>
<affiliation><nlm:aff id="aff4"><institution>INSERM U622</institution>
, Paris,<country>France</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Jens, Jackie K" sort="Jens, Jackie K" uniqKey="Jens J" first="Jackie K" last="Jens">Jackie K. Jens</name>
<affiliation><nlm:aff id="aff1"><institution>Department of Animal Science and the Center for Integrated Animal Genomics, Iowa State University</institution>
, Ames, Iowa,<country>USA</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Snella, Elizabeth M" sort="Snella, Elizabeth M" uniqKey="Snella E" first="Elizabeth M" last="Snella">Elizabeth M. Snella</name>
<affiliation><nlm:aff id="aff1"><institution>Department of Animal Science and the Center for Integrated Animal Genomics, Iowa State University</institution>
, Ames, Iowa,<country>USA</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Mohammed, Eman Ea" sort="Mohammed, Eman Ea" uniqKey="Mohammed E" first="Eman Ea" last="Mohammed">Eman Ea Mohammed</name>
<affiliation><nlm:aff id="aff1"><institution>Department of Animal Science and the Center for Integrated Animal Genomics, Iowa State University</institution>
, Ames, Iowa,<country>USA</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Thomson, Christopher B" sort="Thomson, Christopher B" uniqKey="Thomson C" first="Christopher B" last="Thomson">Christopher B. Thomson</name>
<affiliation><nlm:aff id="aff1"><institution>Department of Animal Science and the Center for Integrated Animal Genomics, Iowa State University</institution>
, Ames, Iowa,<country>USA</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Raoul, Sylvie" sort="Raoul, Sylvie" uniqKey="Raoul S" first="Sylvie" last="Raoul">Sylvie Raoul</name>
<affiliation><nlm:aff id="aff5"><institution>Service de Neurochirurgie, CHU Nord</institution>
, Nantes,<country>France</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Joussemet, Beatrice" sort="Joussemet, Beatrice" uniqKey="Joussemet B" first="Béatrice" last="Joussemet">Béatrice Joussemet</name>
<affiliation><nlm:aff id="aff6"><institution>Centre de Boisbonne, Ecole Nationale Vétérinaire, Agroalimentaire et de l'Alimentation Nantes-Atlantiques (Oniris)</institution>
, Nantes,<country>France</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Roux, Francoise" sort="Roux, Francoise" uniqKey="Roux F" first="Françoise" last="Roux">Françoise Roux</name>
<affiliation><nlm:aff id="aff6"><institution>Centre de Boisbonne, Ecole Nationale Vétérinaire, Agroalimentaire et de l'Alimentation Nantes-Atlantiques (Oniris)</institution>
, Nantes,<country>France</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Cherel, Yan" sort="Cherel, Yan" uniqKey="Cherel Y" first="Yan" last="Chérel">Yan Chérel</name>
<affiliation><nlm:aff id="aff7"><institution>UMR INRA 703, Ecole Nationale Vétérinaire, Agroalimentaire et de l'Alimentation Nantes-Atlantiques (Oniris)</institution>
, Nantes,<country>France</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Lajat, Yaouen" sort="Lajat, Yaouen" uniqKey="Lajat Y" first="Yaouen" last="Lajat">Yaouen Lajat</name>
<affiliation><nlm:aff id="aff5"><institution>Service de Neurochirurgie, CHU Nord</institution>
, Nantes,<country>France</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Piraud, Monique" sort="Piraud, Monique" uniqKey="Piraud M" first="Monique" last="Piraud">Monique Piraud</name>
<affiliation><nlm:aff id="aff8"><institution>Maladies Héréditaire du Métabolisme et Dépistage Néonatal, Centre de Biologie et de Pathologie, Groupement Hospitalier Est, CBPE</institution>
, Bron,<country>France</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Benchaouir, Rachid" sort="Benchaouir, Rachid" uniqKey="Benchaouir R" first="Rachid" last="Benchaouir">Rachid Benchaouir</name>
<affiliation><nlm:aff id="aff9"><institution>Amsterdam Molecular Therapeutics (AMT)</institution>
, Amsterdam,<country>The Netherlands</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Hermening, Stephan" sort="Hermening, Stephan" uniqKey="Hermening S" first="Stephan" last="Hermening">Stephan Hermening</name>
<affiliation><nlm:aff id="aff9"><institution>Amsterdam Molecular Therapeutics (AMT)</institution>
, Amsterdam,<country>The Netherlands</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Petry, Harald" sort="Petry, Harald" uniqKey="Petry H" first="Harald" last="Petry">Harald Petry</name>
<affiliation><nlm:aff id="aff9"><institution>Amsterdam Molecular Therapeutics (AMT)</institution>
, Amsterdam,<country>The Netherlands</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Froissart, Roseline" sort="Froissart, Roseline" uniqKey="Froissart R" first="Roseline" last="Froissart">Roseline Froissart</name>
<affiliation><nlm:aff id="aff8"><institution>Maladies Héréditaire du Métabolisme et Dépistage Néonatal, Centre de Biologie et de Pathologie, Groupement Hospitalier Est, CBPE</institution>
, Bron,<country>France</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Tardieu, Marc" sort="Tardieu, Marc" uniqKey="Tardieu M" first="Marc" last="Tardieu">Marc Tardieu</name>
<affiliation><nlm:aff id="aff10"><institution>Service de Neurologie Pédiatrique, Hôpital Bicêtre, Assistance Publique/Hôpitaux de Paris, INSERM U109</institution>
, le Kremlin-Bicêtre,<country>France</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Ciron, Carine" sort="Ciron, Carine" uniqKey="Ciron C" first="Carine" last="Ciron">Carine Ciron</name>
<affiliation><nlm:aff id="aff11"><institution>INSERM U649, Université de Nantes</institution>
, Nantes,<country>France</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Moullier, Philippe" sort="Moullier, Philippe" uniqKey="Moullier P" first="Philippe" last="Moullier">Philippe Moullier</name>
<affiliation><nlm:aff id="aff11"><institution>INSERM U649, Université de Nantes</institution>
, Nantes,<country>France</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Parkes, Jennifer" sort="Parkes, Jennifer" uniqKey="Parkes J" first="Jennifer" last="Parkes">Jennifer Parkes</name>
<affiliation><nlm:aff id="aff2"><institution>Department of Veterinary Clinical Science, Iowa State University</institution>
, Ames, Iowa,<country>USA</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Kline, Karen L" sort="Kline, Karen L" uniqKey="Kline K" first="Karen L" last="Kline">Karen L. Kline</name>
<affiliation><nlm:aff id="aff2"><institution>Department of Veterinary Clinical Science, Iowa State University</institution>
, Ames, Iowa,<country>USA</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Maire, Irene" sort="Maire, Irene" uniqKey="Maire I" first="Irène" last="Maire">Irène Maire</name>
<affiliation><nlm:aff id="aff8"><institution>Maladies Héréditaire du Métabolisme et Dépistage Néonatal, Centre de Biologie et de Pathologie, Groupement Hospitalier Est, CBPE</institution>
, Bron,<country>France</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Vanier, Marie Therese" sort="Vanier, Marie Therese" uniqKey="Vanier M" first="Marie-Thérèse" last="Vanier">Marie-Thérèse Vanier</name>
<affiliation><nlm:aff id="aff12"><institution>INSERM U820, Faculté de Médecine Laënnec</institution>
, Lyon,<country>France</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Heard, Jean Michel" sort="Heard, Jean Michel" uniqKey="Heard J" first="Jean-Michel" last="Heard">Jean-Michel Heard</name>
<affiliation><nlm:aff id="aff3"><institution>Institut Pasteur, Unité Rétrovirus et Transfert Génétique, Département de Neuroscience</institution>
, Paris,<country>France</country>
</nlm:aff>
</affiliation>
<affiliation><nlm:aff id="aff4"><institution>INSERM U622</institution>
, Paris,<country>France</country>
</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Colle, Marie Anne" sort="Colle, Marie Anne" uniqKey="Colle M" first="Marie-Anne" last="Colle">Marie-Anne Colle</name>
<affiliation><nlm:aff id="aff7"><institution>UMR INRA 703, Ecole Nationale Vétérinaire, Agroalimentaire et de l'Alimentation Nantes-Atlantiques (Oniris)</institution>
, Nantes,<country>France</country>
</nlm:aff>
</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="2010">2010</date>
</imprint>
</series>
</biblStruct>
</sourceDesc>
</fileDesc>
<profileDesc><textClass></textClass>
</profileDesc>
</teiHeader>
<front><div type="abstract" xml:lang="en"><p>Recent trials in patients with neurodegenerative diseases documented the safety of gene therapy based on adeno-associated virus (AAV) vectors deposited into the brain. Inborn errors of the metabolism are the most frequent causes of neurodegeneration in pre-adulthood. In Sanfilippo syndrome, a lysosomal storage disease in which heparan sulfate oligosaccharides accumulate, the onset of clinical manifestation is before 5 years. Studies in the mouse model showed that gene therapy providing the missing enzyme α-<italic>N</italic>
-acetyl-glucosaminidase to brain cells prevents neurodegeneration and improves behavior. We now document safety and efficacy in affected dogs. Animals received eight deposits of a serotype 5 AAV vector, including vector prepared in insect Sf9 cells. As shown previously in dogs with the closely related Hurler syndrome, immunosuppression was necessary to prevent neuroinflammation and elimination of transduced cells. In immunosuppressed dogs, vector was efficiently delivered throughout the brain, induced α-<italic>N</italic>
-acetyl-glucosaminidase production, cleared stored compounds and storage lesions. The suitability of the procedure for clinical application was further assessed in Hurler dogs, providing information on reproducibility, tolerance, appropriate vector type and dosage, and optimal age for treatment in a total number of 25 treated dogs. Results strongly support projects of human trials aimed at assessing this treatment in Sanfilippo syndrome.</p>
</div>
</front>
</TEI>
<pmc article-type="research-article"><pmc-comment>The publisher of this article does not allow downloading of the full text in XML form.</pmc-comment>
<front><journal-meta><journal-id journal-id-type="nlm-ta">Mol Ther</journal-id>
<journal-title-group><journal-title>Molecular Therapy</journal-title>
</journal-title-group>
<issn pub-type="ppub">1525-0016</issn>
<issn pub-type="epub">1525-0024</issn>
<publisher><publisher-name>Nature Publishing Group</publisher-name>
</publisher>
</journal-meta>
<article-meta><article-id pub-id-type="pmid">21139569</article-id>
<article-id pub-id-type="pmc">3034858</article-id>
<article-id pub-id-type="pii">mt2010265</article-id>
<article-id pub-id-type="doi">10.1038/mt.2010.265</article-id>
<article-categories><subj-group subj-group-type="heading"><subject>Original Article</subject>
</subj-group>
</article-categories>
<title-group><article-title>Safe, Efficient, and Reproducible Gene Therapy of the Brain in the Dog Models of Sanfilippo and Hurler Syndromes</article-title>
<alt-title alt-title-type="running">Gene Therapy in Mucopolysaccharidoses</alt-title>
</title-group>
<contrib-group><contrib contrib-type="author"><name><surname>Ellinwood</surname>
<given-names>N Matthew</given-names>
</name>
<xref ref-type="aff" rid="aff1">1</xref>
<xref ref-type="aff" rid="aff2">2</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Ausseil</surname>
<given-names>Jérôme</given-names>
</name>
<xref ref-type="aff" rid="aff3">3</xref>
<xref ref-type="aff" rid="aff4">4</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Desmaris</surname>
<given-names>Nathalie</given-names>
</name>
<xref ref-type="aff" rid="aff3">3</xref>
<xref ref-type="aff" rid="aff4">4</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Bigou</surname>
<given-names>Stéphanie</given-names>
</name>
<xref ref-type="aff" rid="aff3">3</xref>
<xref ref-type="aff" rid="aff4">4</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Liu</surname>
<given-names>Song</given-names>
</name>
<xref ref-type="aff" rid="aff3">3</xref>
<xref ref-type="aff" rid="aff4">4</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Jens</surname>
<given-names>Jackie K</given-names>
</name>
<xref ref-type="aff" rid="aff1">1</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Snella</surname>
<given-names>Elizabeth M</given-names>
</name>
<xref ref-type="aff" rid="aff1">1</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Mohammed</surname>
<given-names>Eman EA</given-names>
</name>
<xref ref-type="aff" rid="aff1">1</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Thomson</surname>
<given-names>Christopher B</given-names>
</name>
<xref ref-type="aff" rid="aff1">1</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Raoul</surname>
<given-names>Sylvie</given-names>
</name>
<xref ref-type="aff" rid="aff5">5</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Joussemet</surname>
<given-names>Béatrice</given-names>
</name>
<xref ref-type="aff" rid="aff6">6</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Roux</surname>
<given-names>Françoise</given-names>
</name>
<xref ref-type="aff" rid="aff6">6</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Chérel</surname>
<given-names>Yan</given-names>
</name>
<xref ref-type="aff" rid="aff7">7</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Lajat</surname>
<given-names>Yaouen</given-names>
</name>
<xref ref-type="aff" rid="aff5">5</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Piraud</surname>
<given-names>Monique</given-names>
</name>
<xref ref-type="aff" rid="aff8">8</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Benchaouir</surname>
<given-names>Rachid</given-names>
</name>
<xref ref-type="aff" rid="aff9">9</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Hermening</surname>
<given-names>Stephan</given-names>
</name>
<xref ref-type="aff" rid="aff9">9</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Petry</surname>
<given-names>Harald</given-names>
</name>
<xref ref-type="aff" rid="aff9">9</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Froissart</surname>
<given-names>Roseline</given-names>
</name>
<xref ref-type="aff" rid="aff8">8</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Tardieu</surname>
<given-names>Marc</given-names>
</name>
<xref ref-type="aff" rid="aff10">10</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Ciron</surname>
<given-names>Carine</given-names>
</name>
<xref ref-type="aff" rid="aff11">11</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Moullier</surname>
<given-names>Philippe</given-names>
</name>
<xref ref-type="aff" rid="aff11">11</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Parkes</surname>
<given-names>Jennifer</given-names>
</name>
<xref ref-type="aff" rid="aff2">2</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Kline</surname>
<given-names>Karen L</given-names>
</name>
<xref ref-type="aff" rid="aff2">2</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Maire</surname>
<given-names>Irène</given-names>
</name>
<xref ref-type="aff" rid="aff8">8</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Vanier</surname>
<given-names>Marie-Thérèse</given-names>
</name>
<xref ref-type="aff" rid="aff12">12</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Heard</surname>
<given-names>Jean-Michel</given-names>
</name>
<xref ref-type="aff" rid="aff3">3</xref>
<xref ref-type="aff" rid="aff4">4</xref>
</contrib>
<contrib contrib-type="author"><name><surname>Colle</surname>
<given-names>Marie-Anne</given-names>
</name>
<xref ref-type="aff" rid="aff7">7</xref>
</contrib>
<aff id="aff1"><label>1</label>
<institution>Department of Animal Science and the Center for Integrated Animal Genomics, Iowa State University</institution>
, Ames, Iowa,<country>USA</country>
</aff>
<aff id="aff2"><label>2</label>
<institution>Department of Veterinary Clinical Science, Iowa State University</institution>
, Ames, Iowa,<country>USA</country>
</aff>
<aff id="aff3"><label>3</label>
<institution>Institut Pasteur, Unité Rétrovirus et Transfert Génétique, Département de Neuroscience</institution>
, Paris,<country>France</country>
</aff>
<aff id="aff4"><label>4</label>
<institution>INSERM U622</institution>
, Paris,<country>France</country>
</aff>
<aff id="aff5"><label>5</label>
<institution>Service de Neurochirurgie, CHU Nord</institution>
, Nantes,<country>France</country>
</aff>
<aff id="aff6"><label>6</label>
<institution>Centre de Boisbonne, Ecole Nationale Vétérinaire, Agroalimentaire et de l'Alimentation Nantes-Atlantiques (Oniris)</institution>
, Nantes,<country>France</country>
</aff>
<aff id="aff7"><label>7</label>
<institution>UMR INRA 703, Ecole Nationale Vétérinaire, Agroalimentaire et de l'Alimentation Nantes-Atlantiques (Oniris)</institution>
, Nantes,<country>France</country>
</aff>
<aff id="aff8"><label>8</label>
<institution>Maladies Héréditaire du Métabolisme et Dépistage Néonatal, Centre de Biologie et de Pathologie, Groupement Hospitalier Est, CBPE</institution>
, Bron,<country>France</country>
</aff>
<aff id="aff9"><label>9</label>
<institution>Amsterdam Molecular Therapeutics (AMT)</institution>
, Amsterdam,<country>The Netherlands</country>
</aff>
<aff id="aff10"><label>10</label>
<institution>Service de Neurologie Pédiatrique, Hôpital Bicêtre, Assistance Publique/Hôpitaux de Paris, INSERM U109</institution>
, le Kremlin-Bicêtre,<country>France</country>
</aff>
<aff id="aff11"><label>11</label>
<institution>INSERM U649, Université de Nantes</institution>
, Nantes,<country>France</country>
</aff>
<aff id="aff12"><label>12</label>
<institution>INSERM U820, Faculté de Médecine Laënnec</institution>
, Lyon,<country>France</country>
</aff>
</contrib-group>
<author-notes><corresp id="caf1"><label>*</label>
Unité Rétrovirus et Transfert Génétique, Département de Neuroscience, Institut Pasteur, 28, rue du Dr Roux, 75015, Paris, France. E-mail: <email>jmheard@pasteur.fr</email>
</corresp>
<fn fn-type="present-address" id="aunote1"><label></label>
<p>The first two authors contributed equally to this work.</p>
</fn>
</author-notes>
<pub-date pub-type="ppub"><month>02</month>
<year>2011</year>
</pub-date>
<pub-date pub-type="epub"><day>07</day>
<month>12</month>
<year>2010</year>
</pub-date>
<volume>19</volume>
<issue>2</issue>
<fpage>251</fpage>
<lpage>259</lpage>
<history><date date-type="received"><day>25</day>
<month>07</month>
<year>2010</year>
</date>
<date date-type="accepted"><day>02</day>
<month>11</month>
<year>2010</year>
</date>
</history>
<permissions><copyright-statement>Copyright © 2011 The American Society of Gene & Cell Therapy</copyright-statement>
<copyright-year>2011</copyright-year>
<copyright-holder>The American Society of Gene & Cell Therapy</copyright-holder>
</permissions>
<abstract><p>Recent trials in patients with neurodegenerative diseases documented the safety of gene therapy based on adeno-associated virus (AAV) vectors deposited into the brain. Inborn errors of the metabolism are the most frequent causes of neurodegeneration in pre-adulthood. In Sanfilippo syndrome, a lysosomal storage disease in which heparan sulfate oligosaccharides accumulate, the onset of clinical manifestation is before 5 years. Studies in the mouse model showed that gene therapy providing the missing enzyme α-<italic>N</italic>
-acetyl-glucosaminidase to brain cells prevents neurodegeneration and improves behavior. We now document safety and efficacy in affected dogs. Animals received eight deposits of a serotype 5 AAV vector, including vector prepared in insect Sf9 cells. As shown previously in dogs with the closely related Hurler syndrome, immunosuppression was necessary to prevent neuroinflammation and elimination of transduced cells. In immunosuppressed dogs, vector was efficiently delivered throughout the brain, induced α-<italic>N</italic>
-acetyl-glucosaminidase production, cleared stored compounds and storage lesions. The suitability of the procedure for clinical application was further assessed in Hurler dogs, providing information on reproducibility, tolerance, appropriate vector type and dosage, and optimal age for treatment in a total number of 25 treated dogs. Results strongly support projects of human trials aimed at assessing this treatment in Sanfilippo syndrome.</p>
</abstract>
</article-meta>
</front>
</pmc>
</record>
Pour manipuler ce document sous Unix (Dilib)
EXPLOR_STEP=$WICRI_ROOT/Wicri/Sante/explor/ParkinsonFranceV1/Data/Pmc/Corpus
HfdSelect -h $EXPLOR_STEP/biblio.hfd -nk 0000070 | SxmlIndent | more
Ou
HfdSelect -h $EXPLOR_AREA/Data/Pmc/Corpus/biblio.hfd -nk 0000070 | SxmlIndent | more
Pour mettre un lien sur cette page dans le réseau Wicri
{{Explor lien |wiki= Wicri/Sante |area= ParkinsonFranceV1 |flux= Pmc |étape= Corpus |type= RBID |clé= |texte= }}
![]() | This area was generated with Dilib version V0.6.29. | ![]() |