Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial.
Identifieur interne : 000591 ( PubMed/Corpus ); précédent : 000590; suivant : 000592Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial.
Auteurs : Stéphane Palfi ; Jean Marc Gurruchaga ; G Scott Ralph ; Helene Lepetit ; Sonia Lavisse ; Philip C. Buttery ; Colin Watts ; James Miskin ; Michelle Kelleher ; Sarah Deeley ; Hirokazu Iwamuro ; Jean Pascal Lefaucheur ; Claire Thiriez ; Gilles Fenelon ; Cherry Lucas ; Pierre Brugières ; Inanna Gabriel ; Kou Abhay ; Xavier Drouot ; Naoki Tani ; Aurelie Kas ; Bijan Ghaleh ; Philippe Le Corvoisier ; Patrice Dolphin ; David P. Breen ; Sarah Mason ; Natalie Valle Guzman ; Nicholas D. Mazarakis ; Pippa A. Radcliffe ; Richard Harrop ; Susan M. Kingsman ; Olivier Rascol ; Stuart Naylor ; Roger A. Barker ; Philippe Hantraye ; Philippe Remy ; Pierre Cesaro ; Kyriacos A. MitrophanousSource :
- Lancet (London, England) [ 1474-547X ] ; 2014.
English descriptors
- KwdEn :
- Aged, Antiparkinson Agents (administration & dosage), Antiparkinson Agents (adverse effects), Dopa Decarboxylase (genetics), Dopamine (biosynthesis), Dopaminergic Neurons (metabolism), Dopaminergic Neurons (virology), Follow-Up Studies, GTP Cyclohydrolase (administration & dosage), GTP Cyclohydrolase (adverse effects), GTP Cyclohydrolase (genetics), Genetic Therapy (adverse effects), Genetic Therapy (methods), Genetic Vectors (administration & dosage), Genetic Vectors (adverse effects), Humans, Infectious Anemia Virus, Equine (genetics), Injections, Intralesional, Male, Middle Aged, Parkinson Disease (therapy), Putamen, Transfection (methods), Transgenes (genetics), Tyrosine 3-Monooxygenase (administration & dosage), Tyrosine 3-Monooxygenase (adverse effects), Tyrosine 3-Monooxygenase (genetics).
- MESH :
- chemical , administration & dosage : Antiparkinson Agents, GTP Cyclohydrolase, Tyrosine 3-Monooxygenase.
- chemical , adverse effects : Antiparkinson Agents, GTP Cyclohydrolase, Tyrosine 3-Monooxygenase.
- chemical , biosynthesis : Dopamine.
- chemical , genetics : Dopa Decarboxylase, GTP Cyclohydrolase, Tyrosine 3-Monooxygenase.
- administration & dosage : Genetic Vectors.
- adverse effects : Genetic Therapy, Genetic Vectors.
- genetics : Infectious Anemia Virus, Equine, Transgenes.
- metabolism : Dopaminergic Neurons.
- methods : Genetic Therapy, Transfection.
- therapy : Parkinson Disease.
- virology : Dopaminergic Neurons.
- Aged, Follow-Up Studies, Humans, Injections, Intralesional, Male, Middle Aged, Putamen.
Abstract
Parkinson's disease is typically treated with oral dopamine replacement therapies; however, long-term treatment leads to motor complications and, occasionally, impulse control disorders caused by intermittent stimulation of dopamine receptors and off-target effects, respectively. We aimed to assess the safety, tolerability, and efficacy of bilateral, intrastriatal delivery of ProSavin, a lentiviral vector-based gene therapy aimed at restoring local and continuous dopamine production in patients with advanced Parkinson's disease.
DOI: 10.1016/S0140-6736(13)61939-X
PubMed: 24412048
Links to Exploration step
pubmed:24412048Le document en format XML
<record><TEI><teiHeader><fileDesc><titleStmt><title xml:lang="en">Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial.</title><author><name sortKey="Palfi, Stephane" sort="Palfi, Stephane" uniqKey="Palfi S" first="Stéphane" last="Palfi">Stéphane Palfi</name><affiliation><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France. Electronic address: stephane.palfi@hmn.aphp.fr.</nlm:affiliation></affiliation></author><author><name sortKey="Gurruchaga, Jean Marc" sort="Gurruchaga, Jean Marc" uniqKey="Gurruchaga J" first="Jean Marc" last="Gurruchaga">Jean Marc Gurruchaga</name><affiliation><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</nlm:affiliation></affiliation></author><author><name sortKey="Ralph, G Scott" sort="Ralph, G Scott" uniqKey="Ralph G" first="G Scott" last="Ralph">G Scott Ralph</name><affiliation><nlm:affiliation>Oxford BioMedica, Oxford, UK.</nlm:affiliation></affiliation></author><author><name sortKey="Lepetit, Helene" sort="Lepetit, Helene" uniqKey="Lepetit H" first="Helene" last="Lepetit">Helene Lepetit</name><affiliation><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</nlm:affiliation></affiliation></author><author><name sortKey="Lavisse, Sonia" sort="Lavisse, Sonia" uniqKey="Lavisse S" first="Sonia" last="Lavisse">Sonia Lavisse</name><affiliation><nlm:affiliation>CEA, DSV I(2)BM, MIRCen and CNRS URA2210, Fontenay-aux-Roses, France.</nlm:affiliation></affiliation></author><author><name sortKey="Buttery, Philip C" sort="Buttery, Philip C" uniqKey="Buttery P" first="Philip C" last="Buttery">Philip C. Buttery</name><affiliation><nlm:affiliation>John van Geest Centre for Brain Repair and Addenbrooke's Hospital, Cambridge, UK.</nlm:affiliation></affiliation></author><author><name sortKey="Watts, Colin" sort="Watts, Colin" uniqKey="Watts C" first="Colin" last="Watts">Colin Watts</name><affiliation><nlm:affiliation>John van Geest Centre for Brain Repair and Addenbrooke's Hospital, Cambridge, UK.</nlm:affiliation></affiliation></author><author><name sortKey="Miskin, James" sort="Miskin, James" uniqKey="Miskin J" first="James" last="Miskin">James Miskin</name><affiliation><nlm:affiliation>Oxford BioMedica, Oxford, UK.</nlm:affiliation></affiliation></author><author><name sortKey="Kelleher, Michelle" sort="Kelleher, Michelle" uniqKey="Kelleher M" first="Michelle" last="Kelleher">Michelle Kelleher</name><affiliation><nlm:affiliation>Oxford BioMedica, Oxford, UK.</nlm:affiliation></affiliation></author><author><name sortKey="Deeley, Sarah" sort="Deeley, Sarah" uniqKey="Deeley S" first="Sarah" last="Deeley">Sarah Deeley</name><affiliation><nlm:affiliation>Oxford BioMedica, Oxford, UK.</nlm:affiliation></affiliation></author><author><name sortKey="Iwamuro, Hirokazu" sort="Iwamuro, Hirokazu" uniqKey="Iwamuro H" first="Hirokazu" last="Iwamuro">Hirokazu Iwamuro</name><affiliation><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</nlm:affiliation></affiliation></author><author><name sortKey="Lefaucheur, Jean Pascal" sort="Lefaucheur, Jean Pascal" uniqKey="Lefaucheur J" first="Jean Pascal" last="Lefaucheur">Jean Pascal Lefaucheur</name><affiliation><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</nlm:affiliation></affiliation></author><author><name sortKey="Thiriez, Claire" sort="Thiriez, Claire" uniqKey="Thiriez C" first="Claire" last="Thiriez">Claire Thiriez</name><affiliation><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</nlm:affiliation></affiliation></author><author><name sortKey="Fenelon, Gilles" sort="Fenelon, Gilles" uniqKey="Fenelon G" first="Gilles" last="Fenelon">Gilles Fenelon</name><affiliation><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France; INSERM U955, E01, Institut de Recherche Biomédicale, Créteil, France.</nlm:affiliation></affiliation></author><author><name sortKey="Lucas, Cherry" sort="Lucas, Cherry" uniqKey="Lucas C" first="Cherry" last="Lucas">Cherry Lucas</name><affiliation><nlm:affiliation>Oxford BioMedica, Oxford, UK.</nlm:affiliation></affiliation></author><author><name sortKey="Brugieres, Pierre" sort="Brugieres, Pierre" uniqKey="Brugieres P" first="Pierre" last="Brugières">Pierre Brugières</name><affiliation><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</nlm:affiliation></affiliation></author><author><name sortKey="Gabriel, Inanna" sort="Gabriel, Inanna" uniqKey="Gabriel I" first="Inanna" last="Gabriel">Inanna Gabriel</name><affiliation><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</nlm:affiliation></affiliation></author><author><name sortKey="Abhay, Kou" sort="Abhay, Kou" uniqKey="Abhay K" first="Kou" last="Abhay">Kou Abhay</name><affiliation><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</nlm:affiliation></affiliation></author><author><name sortKey="Drouot, Xavier" sort="Drouot, Xavier" uniqKey="Drouot X" first="Xavier" last="Drouot">Xavier Drouot</name><affiliation><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</nlm:affiliation></affiliation></author><author><name sortKey="Tani, Naoki" sort="Tani, Naoki" uniqKey="Tani N" first="Naoki" last="Tani">Naoki Tani</name><affiliation><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</nlm:affiliation></affiliation></author><author><name sortKey="Kas, Aurelie" sort="Kas, Aurelie" uniqKey="Kas A" first="Aurelie" last="Kas">Aurelie Kas</name><affiliation><nlm:affiliation>CEA, DSV I(2)BM, MIRCen and CNRS URA2210, Fontenay-aux-Roses, France.</nlm:affiliation></affiliation></author><author><name sortKey="Ghaleh, Bijan" sort="Ghaleh, Bijan" uniqKey="Ghaleh B" first="Bijan" last="Ghaleh">Bijan Ghaleh</name><affiliation><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</nlm:affiliation></affiliation></author><author><name sortKey="Le Corvoisier, Philippe" sort="Le Corvoisier, Philippe" uniqKey="Le Corvoisier P" first="Philippe" last="Le Corvoisier">Philippe Le Corvoisier</name><affiliation><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</nlm:affiliation></affiliation></author><author><name sortKey="Dolphin, Patrice" sort="Dolphin, Patrice" uniqKey="Dolphin P" first="Patrice" last="Dolphin">Patrice Dolphin</name><affiliation><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</nlm:affiliation></affiliation></author><author><name sortKey="Breen, David P" sort="Breen, David P" uniqKey="Breen D" first="David P" last="Breen">David P. Breen</name><affiliation><nlm:affiliation>John van Geest Centre for Brain Repair and Addenbrooke's Hospital, Cambridge, UK.</nlm:affiliation></affiliation></author><author><name sortKey="Mason, Sarah" sort="Mason, Sarah" uniqKey="Mason S" first="Sarah" last="Mason">Sarah Mason</name><affiliation><nlm:affiliation>John van Geest Centre for Brain Repair and Addenbrooke's Hospital, Cambridge, UK.</nlm:affiliation></affiliation></author><author><name sortKey="Guzman, Natalie Valle" sort="Guzman, Natalie Valle" uniqKey="Guzman N" first="Natalie Valle" last="Guzman">Natalie Valle Guzman</name><affiliation><nlm:affiliation>John van Geest Centre for Brain Repair and Addenbrooke's Hospital, Cambridge, UK.</nlm:affiliation></affiliation></author><author><name sortKey="Mazarakis, Nicholas D" sort="Mazarakis, Nicholas D" uniqKey="Mazarakis N" first="Nicholas D" last="Mazarakis">Nicholas D. Mazarakis</name><affiliation><nlm:affiliation>Gene Therapy, Centre of Neuroinflammation and Neurodegeneration, Division of Brain Sciences, Faculty of Medicine, Hammersmith Hospital Campus, London, UK.</nlm:affiliation></affiliation></author><author><name sortKey="Radcliffe, Pippa A" sort="Radcliffe, Pippa A" uniqKey="Radcliffe P" first="Pippa A" last="Radcliffe">Pippa A. Radcliffe</name><affiliation><nlm:affiliation>Oxford BioMedica, Oxford, UK.</nlm:affiliation></affiliation></author><author><name sortKey="Harrop, Richard" sort="Harrop, Richard" uniqKey="Harrop R" first="Richard" last="Harrop">Richard Harrop</name><affiliation><nlm:affiliation>Oxford BioMedica, Oxford, UK.</nlm:affiliation></affiliation></author><author><name sortKey="Kingsman, Susan M" sort="Kingsman, Susan M" uniqKey="Kingsman S" first="Susan M" last="Kingsman">Susan M. Kingsman</name><affiliation><nlm:affiliation>Oxford BioMedica, Oxford, UK.</nlm:affiliation></affiliation></author><author><name sortKey="Rascol, Olivier" sort="Rascol, Olivier" uniqKey="Rascol O" first="Olivier" last="Rascol">Olivier Rascol</name><affiliation><nlm:affiliation>CIC9302 and UMR 825, INSERM and Department of Pharmacology and Neurosciences, University Hospital and University of Toulouse III, Toulouse, France.</nlm:affiliation></affiliation></author><author><name sortKey="Naylor, Stuart" sort="Naylor, Stuart" uniqKey="Naylor S" first="Stuart" last="Naylor">Stuart Naylor</name><affiliation><nlm:affiliation>Oxford BioMedica, Oxford, UK.</nlm:affiliation></affiliation></author><author><name sortKey="Barker, Roger A" sort="Barker, Roger A" uniqKey="Barker R" first="Roger A" last="Barker">Roger A. Barker</name><affiliation><nlm:affiliation>John van Geest Centre for Brain Repair and Addenbrooke's Hospital, Cambridge, UK.</nlm:affiliation></affiliation></author><author><name sortKey="Hantraye, Philippe" sort="Hantraye, Philippe" uniqKey="Hantraye P" first="Philippe" last="Hantraye">Philippe Hantraye</name><affiliation><nlm:affiliation>CEA, DSV I(2)BM, MIRCen and CNRS URA2210, Fontenay-aux-Roses, France.</nlm:affiliation></affiliation></author><author><name sortKey="Remy, Philippe" sort="Remy, Philippe" uniqKey="Remy P" first="Philippe" last="Remy">Philippe Remy</name><affiliation><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France; CEA, DSV I(2)BM, MIRCen and CNRS URA2210, Fontenay-aux-Roses, France.</nlm:affiliation></affiliation></author><author><name sortKey="Cesaro, Pierre" sort="Cesaro, Pierre" uniqKey="Cesaro P" first="Pierre" last="Cesaro">Pierre Cesaro</name><affiliation><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France; INSERM U955, E01, Institut de Recherche Biomédicale, Créteil, France.</nlm:affiliation></affiliation></author><author><name sortKey="Mitrophanous, Kyriacos A" sort="Mitrophanous, Kyriacos A" uniqKey="Mitrophanous K" first="Kyriacos A" last="Mitrophanous">Kyriacos A. Mitrophanous</name><affiliation><nlm:affiliation>Oxford BioMedica, Oxford, UK.</nlm:affiliation></affiliation></author></titleStmt><publicationStmt><idno type="wicri:source">PubMed</idno><date when="2014">2014</date><idno type="RBID">pubmed:24412048</idno><idno type="pmid">24412048</idno><idno type="doi">10.1016/S0140-6736(13)61939-X</idno><idno type="wicri:Area/PubMed/Corpus">000591</idno><idno type="wicri:explorRef" wicri:stream="PubMed" wicri:step="Corpus" wicri:corpus="PubMed">000591</idno></publicationStmt><sourceDesc><biblStruct><analytic><title xml:lang="en">Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial.</title><author><name sortKey="Palfi, Stephane" sort="Palfi, Stephane" uniqKey="Palfi S" first="Stéphane" last="Palfi">Stéphane Palfi</name><affiliation><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France. Electronic address: stephane.palfi@hmn.aphp.fr.</nlm:affiliation></affiliation></author><author><name sortKey="Gurruchaga, Jean Marc" sort="Gurruchaga, Jean Marc" uniqKey="Gurruchaga J" first="Jean Marc" last="Gurruchaga">Jean Marc Gurruchaga</name><affiliation><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</nlm:affiliation></affiliation></author><author><name sortKey="Ralph, G Scott" sort="Ralph, G Scott" uniqKey="Ralph G" first="G Scott" last="Ralph">G Scott Ralph</name><affiliation><nlm:affiliation>Oxford BioMedica, Oxford, UK.</nlm:affiliation></affiliation></author><author><name sortKey="Lepetit, Helene" sort="Lepetit, Helene" uniqKey="Lepetit H" first="Helene" last="Lepetit">Helene Lepetit</name><affiliation><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</nlm:affiliation></affiliation></author><author><name sortKey="Lavisse, Sonia" sort="Lavisse, Sonia" uniqKey="Lavisse S" first="Sonia" last="Lavisse">Sonia Lavisse</name><affiliation><nlm:affiliation>CEA, DSV I(2)BM, MIRCen and CNRS URA2210, Fontenay-aux-Roses, France.</nlm:affiliation></affiliation></author><author><name sortKey="Buttery, Philip C" sort="Buttery, Philip C" uniqKey="Buttery P" first="Philip C" last="Buttery">Philip C. Buttery</name><affiliation><nlm:affiliation>John van Geest Centre for Brain Repair and Addenbrooke's Hospital, Cambridge, UK.</nlm:affiliation></affiliation></author><author><name sortKey="Watts, Colin" sort="Watts, Colin" uniqKey="Watts C" first="Colin" last="Watts">Colin Watts</name><affiliation><nlm:affiliation>John van Geest Centre for Brain Repair and Addenbrooke's Hospital, Cambridge, UK.</nlm:affiliation></affiliation></author><author><name sortKey="Miskin, James" sort="Miskin, James" uniqKey="Miskin J" first="James" last="Miskin">James Miskin</name><affiliation><nlm:affiliation>Oxford BioMedica, Oxford, UK.</nlm:affiliation></affiliation></author><author><name sortKey="Kelleher, Michelle" sort="Kelleher, Michelle" uniqKey="Kelleher M" first="Michelle" last="Kelleher">Michelle Kelleher</name><affiliation><nlm:affiliation>Oxford BioMedica, Oxford, UK.</nlm:affiliation></affiliation></author><author><name sortKey="Deeley, Sarah" sort="Deeley, Sarah" uniqKey="Deeley S" first="Sarah" last="Deeley">Sarah Deeley</name><affiliation><nlm:affiliation>Oxford BioMedica, Oxford, UK.</nlm:affiliation></affiliation></author><author><name sortKey="Iwamuro, Hirokazu" sort="Iwamuro, Hirokazu" uniqKey="Iwamuro H" first="Hirokazu" last="Iwamuro">Hirokazu Iwamuro</name><affiliation><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</nlm:affiliation></affiliation></author><author><name sortKey="Lefaucheur, Jean Pascal" sort="Lefaucheur, Jean Pascal" uniqKey="Lefaucheur J" first="Jean Pascal" last="Lefaucheur">Jean Pascal Lefaucheur</name><affiliation><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</nlm:affiliation></affiliation></author><author><name sortKey="Thiriez, Claire" sort="Thiriez, Claire" uniqKey="Thiriez C" first="Claire" last="Thiriez">Claire Thiriez</name><affiliation><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</nlm:affiliation></affiliation></author><author><name sortKey="Fenelon, Gilles" sort="Fenelon, Gilles" uniqKey="Fenelon G" first="Gilles" last="Fenelon">Gilles Fenelon</name><affiliation><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France; INSERM U955, E01, Institut de Recherche Biomédicale, Créteil, France.</nlm:affiliation></affiliation></author><author><name sortKey="Lucas, Cherry" sort="Lucas, Cherry" uniqKey="Lucas C" first="Cherry" last="Lucas">Cherry Lucas</name><affiliation><nlm:affiliation>Oxford BioMedica, Oxford, UK.</nlm:affiliation></affiliation></author><author><name sortKey="Brugieres, Pierre" sort="Brugieres, Pierre" uniqKey="Brugieres P" first="Pierre" last="Brugières">Pierre Brugières</name><affiliation><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</nlm:affiliation></affiliation></author><author><name sortKey="Gabriel, Inanna" sort="Gabriel, Inanna" uniqKey="Gabriel I" first="Inanna" last="Gabriel">Inanna Gabriel</name><affiliation><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</nlm:affiliation></affiliation></author><author><name sortKey="Abhay, Kou" sort="Abhay, Kou" uniqKey="Abhay K" first="Kou" last="Abhay">Kou Abhay</name><affiliation><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</nlm:affiliation></affiliation></author><author><name sortKey="Drouot, Xavier" sort="Drouot, Xavier" uniqKey="Drouot X" first="Xavier" last="Drouot">Xavier Drouot</name><affiliation><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</nlm:affiliation></affiliation></author><author><name sortKey="Tani, Naoki" sort="Tani, Naoki" uniqKey="Tani N" first="Naoki" last="Tani">Naoki Tani</name><affiliation><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</nlm:affiliation></affiliation></author><author><name sortKey="Kas, Aurelie" sort="Kas, Aurelie" uniqKey="Kas A" first="Aurelie" last="Kas">Aurelie Kas</name><affiliation><nlm:affiliation>CEA, DSV I(2)BM, MIRCen and CNRS URA2210, Fontenay-aux-Roses, France.</nlm:affiliation></affiliation></author><author><name sortKey="Ghaleh, Bijan" sort="Ghaleh, Bijan" uniqKey="Ghaleh B" first="Bijan" last="Ghaleh">Bijan Ghaleh</name><affiliation><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</nlm:affiliation></affiliation></author><author><name sortKey="Le Corvoisier, Philippe" sort="Le Corvoisier, Philippe" uniqKey="Le Corvoisier P" first="Philippe" last="Le Corvoisier">Philippe Le Corvoisier</name><affiliation><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</nlm:affiliation></affiliation></author><author><name sortKey="Dolphin, Patrice" sort="Dolphin, Patrice" uniqKey="Dolphin P" first="Patrice" last="Dolphin">Patrice Dolphin</name><affiliation><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</nlm:affiliation></affiliation></author><author><name sortKey="Breen, David P" sort="Breen, David P" uniqKey="Breen D" first="David P" last="Breen">David P. Breen</name><affiliation><nlm:affiliation>John van Geest Centre for Brain Repair and Addenbrooke's Hospital, Cambridge, UK.</nlm:affiliation></affiliation></author><author><name sortKey="Mason, Sarah" sort="Mason, Sarah" uniqKey="Mason S" first="Sarah" last="Mason">Sarah Mason</name><affiliation><nlm:affiliation>John van Geest Centre for Brain Repair and Addenbrooke's Hospital, Cambridge, UK.</nlm:affiliation></affiliation></author><author><name sortKey="Guzman, Natalie Valle" sort="Guzman, Natalie Valle" uniqKey="Guzman N" first="Natalie Valle" last="Guzman">Natalie Valle Guzman</name><affiliation><nlm:affiliation>John van Geest Centre for Brain Repair and Addenbrooke's Hospital, Cambridge, UK.</nlm:affiliation></affiliation></author><author><name sortKey="Mazarakis, Nicholas D" sort="Mazarakis, Nicholas D" uniqKey="Mazarakis N" first="Nicholas D" last="Mazarakis">Nicholas D. Mazarakis</name><affiliation><nlm:affiliation>Gene Therapy, Centre of Neuroinflammation and Neurodegeneration, Division of Brain Sciences, Faculty of Medicine, Hammersmith Hospital Campus, London, UK.</nlm:affiliation></affiliation></author><author><name sortKey="Radcliffe, Pippa A" sort="Radcliffe, Pippa A" uniqKey="Radcliffe P" first="Pippa A" last="Radcliffe">Pippa A. Radcliffe</name><affiliation><nlm:affiliation>Oxford BioMedica, Oxford, UK.</nlm:affiliation></affiliation></author><author><name sortKey="Harrop, Richard" sort="Harrop, Richard" uniqKey="Harrop R" first="Richard" last="Harrop">Richard Harrop</name><affiliation><nlm:affiliation>Oxford BioMedica, Oxford, UK.</nlm:affiliation></affiliation></author><author><name sortKey="Kingsman, Susan M" sort="Kingsman, Susan M" uniqKey="Kingsman S" first="Susan M" last="Kingsman">Susan M. Kingsman</name><affiliation><nlm:affiliation>Oxford BioMedica, Oxford, UK.</nlm:affiliation></affiliation></author><author><name sortKey="Rascol, Olivier" sort="Rascol, Olivier" uniqKey="Rascol O" first="Olivier" last="Rascol">Olivier Rascol</name><affiliation><nlm:affiliation>CIC9302 and UMR 825, INSERM and Department of Pharmacology and Neurosciences, University Hospital and University of Toulouse III, Toulouse, France.</nlm:affiliation></affiliation></author><author><name sortKey="Naylor, Stuart" sort="Naylor, Stuart" uniqKey="Naylor S" first="Stuart" last="Naylor">Stuart Naylor</name><affiliation><nlm:affiliation>Oxford BioMedica, Oxford, UK.</nlm:affiliation></affiliation></author><author><name sortKey="Barker, Roger A" sort="Barker, Roger A" uniqKey="Barker R" first="Roger A" last="Barker">Roger A. Barker</name><affiliation><nlm:affiliation>John van Geest Centre for Brain Repair and Addenbrooke's Hospital, Cambridge, UK.</nlm:affiliation></affiliation></author><author><name sortKey="Hantraye, Philippe" sort="Hantraye, Philippe" uniqKey="Hantraye P" first="Philippe" last="Hantraye">Philippe Hantraye</name><affiliation><nlm:affiliation>CEA, DSV I(2)BM, MIRCen and CNRS URA2210, Fontenay-aux-Roses, France.</nlm:affiliation></affiliation></author><author><name sortKey="Remy, Philippe" sort="Remy, Philippe" uniqKey="Remy P" first="Philippe" last="Remy">Philippe Remy</name><affiliation><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France; CEA, DSV I(2)BM, MIRCen and CNRS URA2210, Fontenay-aux-Roses, France.</nlm:affiliation></affiliation></author><author><name sortKey="Cesaro, Pierre" sort="Cesaro, Pierre" uniqKey="Cesaro P" first="Pierre" last="Cesaro">Pierre Cesaro</name><affiliation><nlm:affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France; INSERM U955, E01, Institut de Recherche Biomédicale, Créteil, France.</nlm:affiliation></affiliation></author><author><name sortKey="Mitrophanous, Kyriacos A" sort="Mitrophanous, Kyriacos A" uniqKey="Mitrophanous K" first="Kyriacos A" last="Mitrophanous">Kyriacos A. Mitrophanous</name><affiliation><nlm:affiliation>Oxford BioMedica, Oxford, UK.</nlm:affiliation></affiliation></author></analytic><series><title level="j">Lancet (London, England)</title><idno type="eISSN">1474-547X</idno><imprint><date when="2014" type="published">2014</date></imprint></series></biblStruct></sourceDesc></fileDesc><profileDesc><textClass><keywords scheme="KwdEn" xml:lang="en"><term>Aged</term><term>Antiparkinson Agents (administration & dosage)</term><term>Antiparkinson Agents (adverse effects)</term><term>Dopa Decarboxylase (genetics)</term><term>Dopamine (biosynthesis)</term><term>Dopaminergic Neurons (metabolism)</term><term>Dopaminergic Neurons (virology)</term><term>Follow-Up Studies</term><term>GTP Cyclohydrolase (administration & dosage)</term><term>GTP Cyclohydrolase (adverse effects)</term><term>GTP Cyclohydrolase (genetics)</term><term>Genetic Therapy (adverse effects)</term><term>Genetic Therapy (methods)</term><term>Genetic Vectors (administration & dosage)</term><term>Genetic Vectors (adverse effects)</term><term>Humans</term><term>Infectious Anemia Virus, Equine (genetics)</term><term>Injections, Intralesional</term><term>Male</term><term>Middle Aged</term><term>Parkinson Disease (therapy)</term><term>Putamen</term><term>Transfection (methods)</term><term>Transgenes (genetics)</term><term>Tyrosine 3-Monooxygenase (administration & dosage)</term><term>Tyrosine 3-Monooxygenase (adverse effects)</term><term>Tyrosine 3-Monooxygenase (genetics)</term></keywords><keywords scheme="MESH" type="chemical" qualifier="administration & dosage" xml:lang="en"><term>Antiparkinson Agents</term><term>GTP Cyclohydrolase</term><term>Tyrosine 3-Monooxygenase</term></keywords><keywords scheme="MESH" type="chemical" qualifier="adverse effects" xml:lang="en"><term>Antiparkinson Agents</term><term>GTP Cyclohydrolase</term><term>Tyrosine 3-Monooxygenase</term></keywords><keywords scheme="MESH" type="chemical" qualifier="biosynthesis" xml:lang="en"><term>Dopamine</term></keywords><keywords scheme="MESH" type="chemical" qualifier="genetics" xml:lang="en"><term>Dopa Decarboxylase</term><term>GTP Cyclohydrolase</term><term>Tyrosine 3-Monooxygenase</term></keywords><keywords scheme="MESH" qualifier="administration & dosage" xml:lang="en"><term>Genetic Vectors</term></keywords><keywords scheme="MESH" qualifier="adverse effects" xml:lang="en"><term>Genetic Therapy</term><term>Genetic Vectors</term></keywords><keywords scheme="MESH" qualifier="genetics" xml:lang="en"><term>Infectious Anemia Virus, Equine</term><term>Transgenes</term></keywords><keywords scheme="MESH" qualifier="metabolism" xml:lang="en"><term>Dopaminergic Neurons</term></keywords><keywords scheme="MESH" qualifier="methods" xml:lang="en"><term>Genetic Therapy</term><term>Transfection</term></keywords><keywords scheme="MESH" qualifier="therapy" xml:lang="en"><term>Parkinson Disease</term></keywords><keywords scheme="MESH" qualifier="virology" xml:lang="en"><term>Dopaminergic Neurons</term></keywords><keywords scheme="MESH" xml:lang="en"><term>Aged</term><term>Follow-Up Studies</term><term>Humans</term><term>Injections, Intralesional</term><term>Male</term><term>Middle Aged</term><term>Putamen</term></keywords></textClass></profileDesc></teiHeader><front><div type="abstract" xml:lang="en">Parkinson's disease is typically treated with oral dopamine replacement therapies; however, long-term treatment leads to motor complications and, occasionally, impulse control disorders caused by intermittent stimulation of dopamine receptors and off-target effects, respectively. We aimed to assess the safety, tolerability, and efficacy of bilateral, intrastriatal delivery of ProSavin, a lentiviral vector-based gene therapy aimed at restoring local and continuous dopamine production in patients with advanced Parkinson's disease.</div></front></TEI><pubmed><MedlineCitation Status="MEDLINE" Owner="NLM"><PMID Version="1">24412048</PMID><DateCreated><Year>2014</Year><Month>03</Month><Day>31</Day></DateCreated><DateCompleted><Year>2014</Year><Month>04</Month><Day>15</Day></DateCompleted><DateRevised><Year>2015</Year><Month>06</Month><Day>16</Day></DateRevised><Article PubModel="Print-Electronic"><Journal><ISSN IssnType="Electronic">1474-547X</ISSN><JournalIssue CitedMedium="Internet"><Volume>383</Volume><Issue>9923</Issue><PubDate><Year>2014</Year><Month>Mar</Month><Day>29</Day></PubDate></JournalIssue><Title>Lancet (London, England)</Title><ISOAbbreviation>Lancet</ISOAbbreviation></Journal><ArticleTitle>Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial.</ArticleTitle><Pagination><MedlinePgn>1138-46</MedlinePgn></Pagination><ELocationID EIdType="doi" ValidYN="Y">10.1016/S0140-6736(13)61939-X</ELocationID><ELocationID EIdType="pii" ValidYN="Y">S0140-6736(13)61939-X</ELocationID><Abstract><AbstractText Label="BACKGROUND" NlmCategory="BACKGROUND">Parkinson's disease is typically treated with oral dopamine replacement therapies; however, long-term treatment leads to motor complications and, occasionally, impulse control disorders caused by intermittent stimulation of dopamine receptors and off-target effects, respectively. We aimed to assess the safety, tolerability, and efficacy of bilateral, intrastriatal delivery of ProSavin, a lentiviral vector-based gene therapy aimed at restoring local and continuous dopamine production in patients with advanced Parkinson's disease.</AbstractText><AbstractText Label="METHODS" NlmCategory="METHODS">We undertook a phase 1/2 open-label trial with 12-month follow-up at two study sites (France and UK) to assess the safety and efficacy of ProSavin after bilateral injection into the putamen of patients with Parkinson's disease. All patients were then enrolled in a separate open-label follow-up study of long-term safety. Three doses were assessed in separate cohorts: low dose (1·9×10(7) transducing units [TU]); mid dose (4·0×10(7) TU); and high dose (1×10(8) TU). Inclusion criteria were age 48-65 years, disease duration 5 years or longer, motor fluctuations, and 50% or higher motor response to oral dopaminergic therapy. The primary endpoints of the phase 1/2 study were the number and severity of adverse events associated with ProSavin and motor responses as assessed with Unified Parkinson's Disease Rating Scale (UPDRS) part III (off medication) scores, at 6 months after vector administration. Both trials are registered at ClinicalTrials.gov, NCT00627588 and NCT01856439.</AbstractText><AbstractText Label="FINDINGS" NlmCategory="RESULTS">15 patients received ProSavin and were followed up (three at low dose, six mid dose, six high dose). During the first 12 months of follow-up, 54 drug-related adverse events were reported (51 mild, three moderate). Most common were increased on-medication dyskinesias (20 events, 11 patients) and on-off phenomena (12 events, nine patients). No serious adverse events related to the study drug or surgical procedure were reported. A significant improvement in mean UPDRS part III motor scores off medication was recorded in all patients at 6 months (mean score 38 [SD 9] vs 26 [8], n=15, p=0·0001) and 12 months (38 vs 27 [8]; n=15, p=0·0001) compared with baseline.</AbstractText><AbstractText Label="INTERPRETATION" NlmCategory="CONCLUSIONS">ProSavin was safe and well tolerated in patients with advanced Parkinson's disease. Improvement in motor behaviour was observed in all patients.</AbstractText><AbstractText Label="FUNDING" NlmCategory="BACKGROUND">Oxford BioMedica.</AbstractText><CopyrightInformation>Copyright © 2014 Elsevier Ltd. All rights reserved.</CopyrightInformation></Abstract><AuthorList CompleteYN="Y"><Author ValidYN="Y"><LastName>Palfi</LastName><ForeName>Stéphane</ForeName><Initials>S</Initials><AffiliationInfo><Affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France. Electronic address: stephane.palfi@hmn.aphp.fr.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Gurruchaga</LastName><ForeName>Jean Marc</ForeName><Initials>JM</Initials><AffiliationInfo><Affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Ralph</LastName><ForeName>G Scott</ForeName><Initials>GS</Initials><AffiliationInfo><Affiliation>Oxford BioMedica, Oxford, UK.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Lepetit</LastName><ForeName>Helene</ForeName><Initials>H</Initials><AffiliationInfo><Affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Lavisse</LastName><ForeName>Sonia</ForeName><Initials>S</Initials><AffiliationInfo><Affiliation>CEA, DSV I(2)BM, MIRCen and CNRS URA2210, Fontenay-aux-Roses, France.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Buttery</LastName><ForeName>Philip C</ForeName><Initials>PC</Initials><AffiliationInfo><Affiliation>John van Geest Centre for Brain Repair and Addenbrooke's Hospital, Cambridge, UK.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Watts</LastName><ForeName>Colin</ForeName><Initials>C</Initials><AffiliationInfo><Affiliation>John van Geest Centre for Brain Repair and Addenbrooke's Hospital, Cambridge, UK.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Miskin</LastName><ForeName>James</ForeName><Initials>J</Initials><AffiliationInfo><Affiliation>Oxford BioMedica, Oxford, UK.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Kelleher</LastName><ForeName>Michelle</ForeName><Initials>M</Initials><AffiliationInfo><Affiliation>Oxford BioMedica, Oxford, UK.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Deeley</LastName><ForeName>Sarah</ForeName><Initials>S</Initials><AffiliationInfo><Affiliation>Oxford BioMedica, Oxford, UK.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Iwamuro</LastName><ForeName>Hirokazu</ForeName><Initials>H</Initials><AffiliationInfo><Affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Lefaucheur</LastName><ForeName>Jean Pascal</ForeName><Initials>JP</Initials><AffiliationInfo><Affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Thiriez</LastName><ForeName>Claire</ForeName><Initials>C</Initials><AffiliationInfo><Affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Fenelon</LastName><ForeName>Gilles</ForeName><Initials>G</Initials><AffiliationInfo><Affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France; INSERM U955, E01, Institut de Recherche Biomédicale, Créteil, France.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Lucas</LastName><ForeName>Cherry</ForeName><Initials>C</Initials><AffiliationInfo><Affiliation>Oxford BioMedica, Oxford, UK.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Brugières</LastName><ForeName>Pierre</ForeName><Initials>P</Initials><AffiliationInfo><Affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Gabriel</LastName><ForeName>Inanna</ForeName><Initials>I</Initials><AffiliationInfo><Affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Abhay</LastName><ForeName>Kou</ForeName><Initials>K</Initials><AffiliationInfo><Affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Drouot</LastName><ForeName>Xavier</ForeName><Initials>X</Initials><AffiliationInfo><Affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Tani</LastName><ForeName>Naoki</ForeName><Initials>N</Initials><AffiliationInfo><Affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Kas</LastName><ForeName>Aurelie</ForeName><Initials>A</Initials><AffiliationInfo><Affiliation>CEA, DSV I(2)BM, MIRCen and CNRS URA2210, Fontenay-aux-Roses, France.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Ghaleh</LastName><ForeName>Bijan</ForeName><Initials>B</Initials><AffiliationInfo><Affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Le Corvoisier</LastName><ForeName>Philippe</ForeName><Initials>P</Initials><AffiliationInfo><Affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Dolphin</LastName><ForeName>Patrice</ForeName><Initials>P</Initials><AffiliationInfo><Affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Breen</LastName><ForeName>David P</ForeName><Initials>DP</Initials><AffiliationInfo><Affiliation>John van Geest Centre for Brain Repair and Addenbrooke's Hospital, Cambridge, UK.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Mason</LastName><ForeName>Sarah</ForeName><Initials>S</Initials><AffiliationInfo><Affiliation>John van Geest Centre for Brain Repair and Addenbrooke's Hospital, Cambridge, UK.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Guzman</LastName><ForeName>Natalie Valle</ForeName><Initials>NV</Initials><AffiliationInfo><Affiliation>John van Geest Centre for Brain Repair and Addenbrooke's Hospital, Cambridge, UK.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Mazarakis</LastName><ForeName>Nicholas D</ForeName><Initials>ND</Initials><AffiliationInfo><Affiliation>Gene Therapy, Centre of Neuroinflammation and Neurodegeneration, Division of Brain Sciences, Faculty of Medicine, Hammersmith Hospital Campus, London, UK.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Radcliffe</LastName><ForeName>Pippa A</ForeName><Initials>PA</Initials><AffiliationInfo><Affiliation>Oxford BioMedica, Oxford, UK.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Harrop</LastName><ForeName>Richard</ForeName><Initials>R</Initials><AffiliationInfo><Affiliation>Oxford BioMedica, Oxford, UK.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Kingsman</LastName><ForeName>Susan M</ForeName><Initials>SM</Initials><AffiliationInfo><Affiliation>Oxford BioMedica, Oxford, UK.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Rascol</LastName><ForeName>Olivier</ForeName><Initials>O</Initials><AffiliationInfo><Affiliation>CIC9302 and UMR 825, INSERM and Department of Pharmacology and Neurosciences, University Hospital and University of Toulouse III, Toulouse, France.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Naylor</LastName><ForeName>Stuart</ForeName><Initials>S</Initials><AffiliationInfo><Affiliation>Oxford BioMedica, Oxford, UK.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Barker</LastName><ForeName>Roger A</ForeName><Initials>RA</Initials><AffiliationInfo><Affiliation>John van Geest Centre for Brain Repair and Addenbrooke's Hospital, Cambridge, UK.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Hantraye</LastName><ForeName>Philippe</ForeName><Initials>P</Initials><AffiliationInfo><Affiliation>CEA, DSV I(2)BM, MIRCen and CNRS URA2210, Fontenay-aux-Roses, France.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Remy</LastName><ForeName>Philippe</ForeName><Initials>P</Initials><AffiliationInfo><Affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France; CEA, DSV I(2)BM, MIRCen and CNRS URA2210, Fontenay-aux-Roses, France.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Cesaro</LastName><ForeName>Pierre</ForeName><Initials>P</Initials><AffiliationInfo><Affiliation>AP-HP, Groupe Hospitalier Henri-Mondor, DHU PePsy, UF Neurochirurgie Fonctionnelle, Neurologie, Neurophysiologie, Anesthésie, Centre d'Investigation Clinique 006, Plateforme de Ressources Biologiques, Créteil, France; Université Paris 12, Faculté de Médecine, Créteil, France; INSERM U955, E01, Institut de Recherche Biomédicale, Créteil, France.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Mitrophanous</LastName><ForeName>Kyriacos A</ForeName><Initials>KA</Initials><AffiliationInfo><Affiliation>Oxford BioMedica, Oxford, UK.</Affiliation></AffiliationInfo></Author></AuthorList><Language>eng</Language><DataBankList CompleteYN="Y"><DataBank><DataBankName>ClinicalTrials.gov</DataBankName><AccessionNumberList><AccessionNumber>NCT00627588</AccessionNumber><AccessionNumber>NCT01856439</AccessionNumber></AccessionNumberList></DataBank></DataBankList><PublicationTypeList><PublicationType UI="D017426">Clinical Trial, Phase I</PublicationType><PublicationType UI="D017427">Clinical Trial, Phase II</PublicationType><PublicationType UI="D016428">Journal Article</PublicationType><PublicationType UI="D016448">Multicenter Study</PublicationType><PublicationType UI="D013485">Research Support, Non-U.S. Gov't</PublicationType></PublicationTypeList><ArticleDate DateType="Electronic"><Year>2014</Year><Month>01</Month><Day>10</Day></ArticleDate></Article><MedlineJournalInfo><Country>England</Country><MedlineTA>Lancet</MedlineTA><NlmUniqueID>2985213R</NlmUniqueID><ISSNLinking>0140-6736</ISSNLinking></MedlineJournalInfo><ChemicalList><Chemical><RegistryNumber>0</RegistryNumber><NameOfSubstance UI="D000978">Antiparkinson Agents</NameOfSubstance></Chemical><Chemical><RegistryNumber>EC 1.14.16.2</RegistryNumber><NameOfSubstance UI="D014446">Tyrosine 3-Monooxygenase</NameOfSubstance></Chemical><Chemical><RegistryNumber>EC 3.5.4.16</RegistryNumber><NameOfSubstance UI="D006136">GTP Cyclohydrolase</NameOfSubstance></Chemical><Chemical><RegistryNumber>EC 4.1.1.-</RegistryNumber><NameOfSubstance UI="D004296">Dopa Decarboxylase</NameOfSubstance></Chemical><Chemical><RegistryNumber>VTD58H1Z2X</RegistryNumber><NameOfSubstance UI="D004298">Dopamine</NameOfSubstance></Chemical></ChemicalList><CitationSubset>AIM</CitationSubset><CitationSubset>IM</CitationSubset><CommentsCorrectionsList><CommentsCorrections RefType="CommentIn"><RefSource>Lancet. 2014 Mar 29;383(9923):1107-9</RefSource><PMID Version="1">24412047</PMID></CommentsCorrections><CommentsCorrections RefType="CommentIn"><RefSource>Nat Rev Neurol. 2014 Apr;10(4):186-7</RefSource><PMID Version="1">24662536</PMID></CommentsCorrections></CommentsCorrectionsList><MeshHeadingList><MeshHeading><DescriptorName UI="D000368" MajorTopicYN="N">Aged</DescriptorName></MeshHeading><MeshHeading><DescriptorName UI="D000978" MajorTopicYN="N">Antiparkinson Agents</DescriptorName><QualifierName UI="Q000008" MajorTopicYN="Y">administration & dosage</QualifierName><QualifierName UI="Q000009" MajorTopicYN="N">adverse effects</QualifierName></MeshHeading><MeshHeading><DescriptorName UI="D004296" MajorTopicYN="N">Dopa Decarboxylase</DescriptorName><QualifierName UI="Q000235" MajorTopicYN="N">genetics</QualifierName></MeshHeading><MeshHeading><DescriptorName UI="D004298" MajorTopicYN="N">Dopamine</DescriptorName><QualifierName UI="Q000096" MajorTopicYN="N">biosynthesis</QualifierName></MeshHeading><MeshHeading><DescriptorName UI="D059290" MajorTopicYN="N">Dopaminergic Neurons</DescriptorName><QualifierName UI="Q000378" MajorTopicYN="N">metabolism</QualifierName><QualifierName UI="Q000821" MajorTopicYN="N">virology</QualifierName></MeshHeading><MeshHeading><DescriptorName UI="D005500" MajorTopicYN="N">Follow-Up Studies</DescriptorName></MeshHeading><MeshHeading><DescriptorName UI="D006136" MajorTopicYN="N">GTP Cyclohydrolase</DescriptorName><QualifierName UI="Q000008" MajorTopicYN="N">administration & dosage</QualifierName><QualifierName UI="Q000009" MajorTopicYN="N">adverse effects</QualifierName><QualifierName UI="Q000235" MajorTopicYN="N">genetics</QualifierName></MeshHeading><MeshHeading><DescriptorName UI="D015316" MajorTopicYN="N">Genetic Therapy</DescriptorName><QualifierName UI="Q000009" MajorTopicYN="N">adverse effects</QualifierName><QualifierName UI="Q000379" MajorTopicYN="Y">methods</QualifierName></MeshHeading><MeshHeading><DescriptorName UI="D005822" MajorTopicYN="N">Genetic Vectors</DescriptorName><QualifierName UI="Q000008" MajorTopicYN="Y">administration & dosage</QualifierName><QualifierName UI="Q000009" MajorTopicYN="N">adverse effects</QualifierName></MeshHeading><MeshHeading><DescriptorName UI="D006801" MajorTopicYN="N">Humans</DescriptorName></MeshHeading><MeshHeading><DescriptorName UI="D004860" MajorTopicYN="N">Infectious Anemia Virus, Equine</DescriptorName><QualifierName UI="Q000235" MajorTopicYN="Y">genetics</QualifierName></MeshHeading><MeshHeading><DescriptorName UI="D015552" MajorTopicYN="N">Injections, Intralesional</DescriptorName></MeshHeading><MeshHeading><DescriptorName UI="D008297" MajorTopicYN="N">Male</DescriptorName></MeshHeading><MeshHeading><DescriptorName UI="D008875" MajorTopicYN="N">Middle Aged</DescriptorName></MeshHeading><MeshHeading><DescriptorName UI="D010300" MajorTopicYN="N">Parkinson Disease</DescriptorName><QualifierName UI="Q000628" MajorTopicYN="Y">therapy</QualifierName></MeshHeading><MeshHeading><DescriptorName UI="D011699" MajorTopicYN="N">Putamen</DescriptorName></MeshHeading><MeshHeading><DescriptorName UI="D014162" MajorTopicYN="N">Transfection</DescriptorName><QualifierName UI="Q000379" MajorTopicYN="Y">methods</QualifierName></MeshHeading><MeshHeading><DescriptorName UI="D019076" MajorTopicYN="N">Transgenes</DescriptorName><QualifierName UI="Q000235" MajorTopicYN="N">genetics</QualifierName></MeshHeading><MeshHeading><DescriptorName UI="D014446" MajorTopicYN="N">Tyrosine 3-Monooxygenase</DescriptorName><QualifierName UI="Q000008" MajorTopicYN="N">administration & dosage</QualifierName><QualifierName UI="Q000009" MajorTopicYN="N">adverse effects</QualifierName><QualifierName UI="Q000235" MajorTopicYN="N">genetics</QualifierName></MeshHeading></MeshHeadingList></MedlineCitation><PubmedData><History><PubMedPubDate PubStatus="entrez"><Year>2014</Year><Month>1</Month><Day>14</Day><Hour>6</Hour><Minute>0</Minute></PubMedPubDate><PubMedPubDate PubStatus="pubmed"><Year>2014</Year><Month>1</Month><Day>15</Day><Hour>6</Hour><Minute>0</Minute></PubMedPubDate><PubMedPubDate PubStatus="medline"><Year>2014</Year><Month>4</Month><Day>16</Day><Hour>6</Hour><Minute>0</Minute></PubMedPubDate></History><PublicationStatus>ppublish</PublicationStatus><ArticleIdList><ArticleId IdType="pubmed">24412048</ArticleId><ArticleId IdType="pii">S0140-6736(13)61939-X</ArticleId><ArticleId IdType="doi">10.1016/S0140-6736(13)61939-X</ArticleId></ArticleIdList></PubmedData></pubmed></record>Pour manipuler ce document sous Unix (Dilib)
EXPLOR_STEP=$WICRI_ROOT/Wicri/Sante/explor/ParkinsonFranceV1/Data/PubMed/Corpus
HfdSelect -h $EXPLOR_STEP/biblio.hfd -nk 000591 | SxmlIndent | more
Ou
HfdSelect -h $EXPLOR_AREA/Data/PubMed/Corpus/biblio.hfd -nk 000591 | SxmlIndent | more
Pour mettre un lien sur cette page dans le réseau Wicri
{{Explor lien
|wiki= Wicri/Sante
|area= ParkinsonFranceV1
|flux= PubMed
|étape= Corpus
|type= RBID
|clé= pubmed:24412048
|texte= Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial.
}}
Pour générer des pages wiki
HfdIndexSelect -h $EXPLOR_AREA/Data/PubMed/Corpus/RBID.i -Sk "pubmed:24412048" \
| HfdSelect -Kh $EXPLOR_AREA/Data/PubMed/Corpus/biblio.hfd \
| NlmPubMed2Wicri -a ParkinsonFranceV1
| This area was generated with Dilib version V0.6.29. |  |