Gene delivery of neurturin to putamen and substantia nigra in Parkinson disease: A double-blind, randomized, controlled trial.
Identifieur interne : 000716 ( Main/Exploration ); précédent : 000715; suivant : 000717Gene delivery of neurturin to putamen and substantia nigra in Parkinson disease: A double-blind, randomized, controlled trial.
Auteurs : C. Warren Olanow [États-Unis] ; Raymond T. Bartus [États-Unis] ; Tiffany L. Baumann [États-Unis] ; Stewart Factor [États-Unis] ; Nicholas Boulis [États-Unis] ; Mark Stacy [États-Unis] ; Dennis A. Turner [États-Unis] ; William Marks [États-Unis] ; Paul Larson [États-Unis] ; Phillip A. Starr [États-Unis] ; Joseph Jankovic [États-Unis] ; Richard Simpson [États-Unis] ; Ray Watts [États-Unis] ; Barton Guthrie [États-Unis] ; Kathleen Poston [États-Unis] ; Jaimie M. Henderson [États-Unis] ; Matthew Stern [États-Unis] ; Gordon Baltuch [États-Unis] ; Christopher G. Goetz [États-Unis] ; Christopher Herzog [États-Unis] ; Jeffrey H. Kordower [États-Unis] ; Ron Alterman [États-Unis] ; Andres M. Lozano [Canada] ; Anthony E. Lang [Canada]Source :
- Annals of neurology [ 1531-8249 ] ; 2015.
English descriptors
- KwdEn :
- Aged, Axonal Transport, Dependovirus, Double-Blind Method, Female, Genetic Therapy (methods), Genetic Vectors (therapeutic use), Humans, Male, Middle Aged, Neurturin (genetics), Parkinson Disease (metabolism), Parkinson Disease (physiopathology), Parkinson Disease (therapy), Putamen (metabolism), Putamen (physiopathology), Substantia Nigra (metabolism), Substantia Nigra (physiopathology), Treatment Outcome.
- MESH :
- chemical , genetics : Neurturin.
- metabolism : Parkinson Disease, Putamen, Substantia Nigra.
- methods : Genetic Therapy.
- physiopathology : Parkinson Disease, Putamen, Substantia Nigra.
- therapeutic use : Genetic Vectors.
- therapy : Parkinson Disease.
- Aged, Axonal Transport, Dependovirus, Double-Blind Method, Female, Humans, Male, Middle Aged, Treatment Outcome.
Abstract
A 12-month double-blind sham-surgery-controlled trial assessing adeno-associated virus type 2 (AAV2)-neurturin injected into the putamen bilaterally failed to meet its primary endpoint, but showed positive results for the primary endpoint in the subgroup of subjects followed for 18 months and for several secondary endpoints. Analysis of postmortem tissue suggested impaired axonal transport of neurturin from putamen to substantia nigra. In the present study, we tested the safety and efficacy of AAV2-neurturin delivered to putamen and substantia nigra.
DOI: 10.1002/ana.24436
PubMed: 26061140
Affiliations:
- Canada, États-Unis
- Alabama, Californie, Caroline du Nord, Géorgie (États-Unis), Illinois, Massachusetts, Ontario, Pennsylvanie, Texas, État de New York
- Houston, Toronto
- Baylor College of Medicine, Université de Toronto
Links toward previous steps (curation, corpus...)
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Le document en format XML
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Starr</name><affiliation wicri:level="2"><nlm:affiliation>Department of Neurological Surgery, University of California, San Francisco, San Francisco, CA.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">Californie</region></placeName><wicri:cityArea>Department of Neurological Surgery, University of California, San Francisco, San Francisco</wicri:cityArea></affiliation></author><author><name sortKey="Jankovic, Joseph" sort="Jankovic, Joseph" uniqKey="Jankovic J" first="Joseph" last="Jankovic">Joseph Jankovic</name><affiliation wicri:level="2"><nlm:affiliation>Department of Neurology, Baylor College of Medicine, Houston, TX.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">Texas</region></placeName><wicri:cityArea>Department of Neurology, Baylor College of Medicine, Houston</wicri:cityArea><placeName><settlement type="city">Houston</settlement><region type="state">Texas</region></placeName><orgName type="university" n="3">Baylor College of Medicine</orgName><placeName><settlement type="city">Houston</settlement><region type="state">Texas</region></placeName><orgName type="university" n="3">Baylor College of Medicine</orgName></affiliation></author><author><name sortKey="Simpson, Richard" sort="Simpson, Richard" uniqKey="Simpson R" first="Richard" last="Simpson">Richard Simpson</name><affiliation wicri:level="2"><nlm:affiliation>Department of Neurosurgery, Baylor College of Medicine, Houston, TX.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">Texas</region></placeName><wicri:cityArea>Department of Neurosurgery, Baylor College of Medicine, Houston</wicri:cityArea></affiliation></author><author><name sortKey="Watts, Ray" sort="Watts, Ray" uniqKey="Watts R" first="Ray" last="Watts">Ray Watts</name><affiliation wicri:level="2"><nlm:affiliation>Department of Neurology, University of Alabama at Birmingham, Birmingham, AL.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">Alabama</region></placeName><wicri:cityArea>Department of Neurology, University of Alabama at Birmingham, Birmingham</wicri:cityArea></affiliation></author><author><name sortKey="Guthrie, Barton" sort="Guthrie, Barton" uniqKey="Guthrie B" first="Barton" last="Guthrie">Barton Guthrie</name><affiliation wicri:level="2"><nlm:affiliation>Department of Neurosurgery, University of Alabama at Birmingham, Birmingham, AL.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">Alabama</region></placeName><wicri:cityArea>Department of Neurosurgery, University of Alabama at Birmingham, Birmingham</wicri:cityArea></affiliation></author><author><name sortKey="Poston, Kathleen" sort="Poston, Kathleen" uniqKey="Poston K" first="Kathleen" last="Poston">Kathleen Poston</name><affiliation wicri:level="2"><nlm:affiliation>Department of Neurology and Neurological Sciences, Stanford University Medical Center, Stanford, CA.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">Californie</region></placeName><wicri:cityArea>Department of Neurology and Neurological Sciences, Stanford University Medical Center, Stanford</wicri:cityArea></affiliation></author><author><name sortKey="Henderson, Jaimie M" sort="Henderson, Jaimie M" uniqKey="Henderson J" first="Jaimie M" last="Henderson">Jaimie M. Henderson</name><affiliation wicri:level="2"><nlm:affiliation>Department of Neurosurgery, Stanford University Medical Center, Stanford, CA.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">Californie</region></placeName><wicri:cityArea>Department of Neurosurgery, Stanford University Medical Center, Stanford</wicri:cityArea></affiliation></author><author><name sortKey="Stern, Matthew" sort="Stern, Matthew" uniqKey="Stern M" first="Matthew" last="Stern">Matthew Stern</name><affiliation wicri:level="2"><nlm:affiliation>Department of Neurology, University of Pennsylvania, Philadelphia, PA.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">Pennsylvanie</region></placeName><wicri:cityArea>Department of Neurology, University of Pennsylvania, Philadelphia</wicri:cityArea></affiliation></author><author><name sortKey="Baltuch, Gordon" sort="Baltuch, Gordon" uniqKey="Baltuch G" first="Gordon" last="Baltuch">Gordon Baltuch</name><affiliation wicri:level="2"><nlm:affiliation>Department of Neurosurgery, University of Pennsylvania, Philadelphia, PA.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">Pennsylvanie</region></placeName><wicri:cityArea>Department of Neurosurgery, University of Pennsylvania, Philadelphia</wicri:cityArea></affiliation></author><author><name sortKey="Goetz, Christopher G" sort="Goetz, Christopher G" uniqKey="Goetz C" first="Christopher G" last="Goetz">Christopher G. Goetz</name><affiliation wicri:level="2"><nlm:affiliation>Department of Neurological Sciences, Rush University Medical Center, Chicago, IL.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">Illinois</region></placeName><wicri:cityArea>Department of Neurological Sciences, Rush University Medical Center, Chicago</wicri:cityArea></affiliation></author><author><name sortKey="Herzog, Christopher" sort="Herzog, Christopher" uniqKey="Herzog C" first="Christopher" last="Herzog">Christopher Herzog</name><affiliation wicri:level="2"><nlm:affiliation>Ceregene, San Diego, CA.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">Californie</region></placeName><wicri:cityArea>Ceregene, San Diego</wicri:cityArea></affiliation></author><author><name sortKey="Kordower, Jeffrey H" sort="Kordower, Jeffrey H" uniqKey="Kordower J" first="Jeffrey H" last="Kordower">Jeffrey H. Kordower</name><affiliation wicri:level="2"><nlm:affiliation>Department of Neurological Sciences, Rush University Medical Center, Chicago, IL.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">Illinois</region></placeName><wicri:cityArea>Department of Neurological Sciences, Rush University Medical Center, Chicago</wicri:cityArea></affiliation></author><author><name sortKey="Alterman, Ron" sort="Alterman, Ron" uniqKey="Alterman R" first="Ron" last="Alterman">Ron Alterman</name><affiliation wicri:level="2"><nlm:affiliation>Beth Israel-Deaconess Medical Center, Department of Neurosurgery, Boston, MA.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">Massachusetts</region></placeName><wicri:cityArea>Beth Israel-Deaconess Medical Center, Department of Neurosurgery, Boston</wicri:cityArea></affiliation></author><author><name sortKey="Lozano, Andres M" sort="Lozano, Andres M" uniqKey="Lozano A" first="Andres M" last="Lozano">Andres M. Lozano</name><affiliation wicri:level="4"><nlm:affiliation>Division of Neurosurgery, Department of Surgery, Toronto Western Hospital, University of Toronto, Toronto, Ontario, Canada.</nlm:affiliation><country xml:lang="fr">Canada</country><wicri:regionArea>Division of Neurosurgery, Department of Surgery, Toronto Western Hospital, University of Toronto, Toronto, Ontario</wicri:regionArea><orgName type="university">Université de Toronto</orgName><placeName><settlement type="city">Toronto</settlement><region type="state">Ontario</region></placeName></affiliation></author><author><name sortKey="Lang, Anthony E" sort="Lang, Anthony E" uniqKey="Lang A" first="Anthony E" last="Lang">Anthony E. 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Warren Olanow</name><affiliation wicri:level="2"><nlm:affiliation>Departments of Neurology and Neuroscience, Mount Sinai School of Medicine, New York, NY.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">État de New York</region></placeName><wicri:cityArea>Departments of Neurology and Neuroscience, Mount Sinai School of Medicine, New York</wicri:cityArea></affiliation></author><author><name sortKey="Bartus, Raymond T" sort="Bartus, Raymond T" uniqKey="Bartus R" first="Raymond T" last="Bartus">Raymond T. Bartus</name><affiliation wicri:level="2"><nlm:affiliation>RTBioconsultants, San Diego, CA.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">Californie</region></placeName><wicri:cityArea>RTBioconsultants, San Diego</wicri:cityArea></affiliation></author><author><name sortKey="Baumann, Tiffany L" sort="Baumann, Tiffany L" uniqKey="Baumann T" first="Tiffany L" last="Baumann">Tiffany L. Baumann</name><affiliation wicri:level="2"><nlm:affiliation>Isis Pharmaceuticals, Carlsbad, CA.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">Californie</region></placeName><wicri:cityArea>Isis Pharmaceuticals, Carlsbad</wicri:cityArea></affiliation></author><author><name sortKey="Factor, Stewart" sort="Factor, Stewart" uniqKey="Factor S" first="Stewart" last="Factor">Stewart Factor</name><affiliation wicri:level="2"><nlm:affiliation>Department of Neurology, Emory University School of Medicine, Atlanta, GA.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">Géorgie (États-Unis)</region></placeName><wicri:cityArea>Department of Neurology, Emory University School of Medicine, Atlanta</wicri:cityArea></affiliation></author><author><name sortKey="Boulis, Nicholas" sort="Boulis, Nicholas" uniqKey="Boulis N" first="Nicholas" last="Boulis">Nicholas Boulis</name><affiliation wicri:level="2"><nlm:affiliation>Department of Neurosurgery, Emory University School of Medicine, Atlanta, GA.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">Géorgie (États-Unis)</region></placeName><wicri:cityArea>Department of Neurosurgery, Emory University School of Medicine, Atlanta</wicri:cityArea></affiliation></author><author><name sortKey="Stacy, Mark" sort="Stacy, Mark" uniqKey="Stacy M" first="Mark" last="Stacy">Mark Stacy</name><affiliation wicri:level="2"><nlm:affiliation>Department of Neurology, Duke University School of Medicine, Durham, NC.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">Caroline du Nord</region></placeName><wicri:cityArea>Department of Neurology, Duke University School of Medicine, Durham</wicri:cityArea></affiliation></author><author><name sortKey="Turner, Dennis A" sort="Turner, Dennis A" uniqKey="Turner D" first="Dennis A" last="Turner">Dennis A. Turner</name><affiliation wicri:level="2"><nlm:affiliation>Department of Neurosurgery, Duke University School of Medicine, Durham, NC.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">Caroline du Nord</region></placeName><wicri:cityArea>Department of Neurosurgery, Duke University School of Medicine, Durham</wicri:cityArea></affiliation></author><author><name sortKey="Marks, William" sort="Marks, William" uniqKey="Marks W" first="William" last="Marks">William Marks</name><affiliation wicri:level="2"><nlm:affiliation>Department of Neurological Surgery, University of California, San Francisco, San Francisco, CA.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">Californie</region></placeName><wicri:cityArea>Department of Neurological Surgery, University of California, San Francisco, San Francisco</wicri:cityArea></affiliation></author><author><name sortKey="Larson, Paul" sort="Larson, Paul" uniqKey="Larson P" first="Paul" last="Larson">Paul Larson</name><affiliation wicri:level="2"><nlm:affiliation>Department of Neurological Surgery, University of California, San Francisco, San Francisco, CA.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">Californie</region></placeName><wicri:cityArea>Department of Neurological Surgery, University of California, San Francisco, San Francisco</wicri:cityArea></affiliation></author><author><name sortKey="Starr, Phillip A" sort="Starr, Phillip A" uniqKey="Starr P" first="Phillip A" last="Starr">Phillip A. Starr</name><affiliation wicri:level="2"><nlm:affiliation>Department of Neurological Surgery, University of California, San Francisco, San Francisco, CA.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">Californie</region></placeName><wicri:cityArea>Department of Neurological Surgery, University of California, San Francisco, San Francisco</wicri:cityArea></affiliation></author><author><name sortKey="Jankovic, Joseph" sort="Jankovic, Joseph" uniqKey="Jankovic J" first="Joseph" last="Jankovic">Joseph Jankovic</name><affiliation wicri:level="2"><nlm:affiliation>Department of Neurology, Baylor College of Medicine, Houston, TX.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">Texas</region></placeName><wicri:cityArea>Department of Neurology, Baylor College of Medicine, Houston</wicri:cityArea><placeName><settlement type="city">Houston</settlement><region type="state">Texas</region></placeName><orgName type="university" n="3">Baylor College of Medicine</orgName><placeName><settlement type="city">Houston</settlement><region type="state">Texas</region></placeName><orgName type="university" n="3">Baylor College of Medicine</orgName></affiliation></author><author><name sortKey="Simpson, Richard" sort="Simpson, Richard" uniqKey="Simpson R" first="Richard" last="Simpson">Richard Simpson</name><affiliation wicri:level="2"><nlm:affiliation>Department of Neurosurgery, Baylor College of Medicine, Houston, TX.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">Texas</region></placeName><wicri:cityArea>Department of Neurosurgery, Baylor College of Medicine, Houston</wicri:cityArea></affiliation></author><author><name sortKey="Watts, Ray" sort="Watts, Ray" uniqKey="Watts R" first="Ray" last="Watts">Ray Watts</name><affiliation wicri:level="2"><nlm:affiliation>Department of Neurology, University of Alabama at Birmingham, Birmingham, AL.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">Alabama</region></placeName><wicri:cityArea>Department of Neurology, University of Alabama at Birmingham, Birmingham</wicri:cityArea></affiliation></author><author><name sortKey="Guthrie, Barton" sort="Guthrie, Barton" uniqKey="Guthrie B" first="Barton" last="Guthrie">Barton Guthrie</name><affiliation wicri:level="2"><nlm:affiliation>Department of Neurosurgery, University of Alabama at Birmingham, Birmingham, AL.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">Alabama</region></placeName><wicri:cityArea>Department of Neurosurgery, University of Alabama at Birmingham, Birmingham</wicri:cityArea></affiliation></author><author><name sortKey="Poston, Kathleen" sort="Poston, Kathleen" uniqKey="Poston K" first="Kathleen" last="Poston">Kathleen Poston</name><affiliation wicri:level="2"><nlm:affiliation>Department of Neurology and Neurological Sciences, Stanford University Medical Center, Stanford, CA.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">Californie</region></placeName><wicri:cityArea>Department of Neurology and Neurological Sciences, Stanford University Medical Center, Stanford</wicri:cityArea></affiliation></author><author><name sortKey="Henderson, Jaimie M" sort="Henderson, Jaimie M" uniqKey="Henderson J" first="Jaimie M" last="Henderson">Jaimie M. Henderson</name><affiliation wicri:level="2"><nlm:affiliation>Department of Neurosurgery, Stanford University Medical Center, Stanford, CA.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">Californie</region></placeName><wicri:cityArea>Department of Neurosurgery, Stanford University Medical Center, Stanford</wicri:cityArea></affiliation></author><author><name sortKey="Stern, Matthew" sort="Stern, Matthew" uniqKey="Stern M" first="Matthew" last="Stern">Matthew Stern</name><affiliation wicri:level="2"><nlm:affiliation>Department of Neurology, University of Pennsylvania, Philadelphia, PA.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">Pennsylvanie</region></placeName><wicri:cityArea>Department of Neurology, University of Pennsylvania, Philadelphia</wicri:cityArea></affiliation></author><author><name sortKey="Baltuch, Gordon" sort="Baltuch, Gordon" uniqKey="Baltuch G" first="Gordon" last="Baltuch">Gordon Baltuch</name><affiliation wicri:level="2"><nlm:affiliation>Department of Neurosurgery, University of Pennsylvania, Philadelphia, PA.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">Pennsylvanie</region></placeName><wicri:cityArea>Department of Neurosurgery, University of Pennsylvania, Philadelphia</wicri:cityArea></affiliation></author><author><name sortKey="Goetz, Christopher G" sort="Goetz, Christopher G" uniqKey="Goetz C" first="Christopher G" last="Goetz">Christopher G. Goetz</name><affiliation wicri:level="2"><nlm:affiliation>Department of Neurological Sciences, Rush University Medical Center, Chicago, IL.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">Illinois</region></placeName><wicri:cityArea>Department of Neurological Sciences, Rush University Medical Center, Chicago</wicri:cityArea></affiliation></author><author><name sortKey="Herzog, Christopher" sort="Herzog, Christopher" uniqKey="Herzog C" first="Christopher" last="Herzog">Christopher Herzog</name><affiliation wicri:level="2"><nlm:affiliation>Ceregene, San Diego, CA.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">Californie</region></placeName><wicri:cityArea>Ceregene, San Diego</wicri:cityArea></affiliation></author><author><name sortKey="Kordower, Jeffrey H" sort="Kordower, Jeffrey H" uniqKey="Kordower J" first="Jeffrey H" last="Kordower">Jeffrey H. Kordower</name><affiliation wicri:level="2"><nlm:affiliation>Department of Neurological Sciences, Rush University Medical Center, Chicago, IL.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">Illinois</region></placeName><wicri:cityArea>Department of Neurological Sciences, Rush University Medical Center, Chicago</wicri:cityArea></affiliation></author><author><name sortKey="Alterman, Ron" sort="Alterman, Ron" uniqKey="Alterman R" first="Ron" last="Alterman">Ron Alterman</name><affiliation wicri:level="2"><nlm:affiliation>Beth Israel-Deaconess Medical Center, Department of Neurosurgery, Boston, MA.</nlm:affiliation><country xml:lang="fr">États-Unis</country><placeName><region type="state">Massachusetts</region></placeName><wicri:cityArea>Beth Israel-Deaconess Medical Center, Department of Neurosurgery, Boston</wicri:cityArea></affiliation></author><author><name sortKey="Lozano, Andres M" sort="Lozano, Andres M" uniqKey="Lozano A" first="Andres M" last="Lozano">Andres M. Lozano</name><affiliation wicri:level="4"><nlm:affiliation>Division of Neurosurgery, Department of Surgery, Toronto Western Hospital, University of Toronto, Toronto, Ontario, Canada.</nlm:affiliation><country xml:lang="fr">Canada</country><wicri:regionArea>Division of Neurosurgery, Department of Surgery, Toronto Western Hospital, University of Toronto, Toronto, Ontario</wicri:regionArea><orgName type="university">Université de Toronto</orgName><placeName><settlement type="city">Toronto</settlement><region type="state">Ontario</region></placeName></affiliation></author><author><name sortKey="Lang, Anthony E" sort="Lang, Anthony E" uniqKey="Lang A" first="Anthony E" last="Lang">Anthony E. Lang</name><affiliation wicri:level="4"><nlm:affiliation>Division of Neurology, Department of Medicine, University of Toronto, Toronto, Ontario, Canada.</nlm:affiliation><country xml:lang="fr">Canada</country><wicri:regionArea>Division of Neurology, Department of Medicine, University of Toronto, Toronto, Ontario</wicri:regionArea><orgName type="university">Université de Toronto</orgName><placeName><settlement type="city">Toronto</settlement><region type="state">Ontario</region></placeName></affiliation></author></analytic><series><title level="j">Annals of neurology</title><idno type="eISSN">1531-8249</idno><imprint><date when="2015" type="published">2015</date></imprint></series></biblStruct></sourceDesc></fileDesc><profileDesc><textClass><keywords scheme="KwdEn" xml:lang="en"><term>Aged</term><term>Axonal Transport</term><term>Dependovirus</term><term>Double-Blind Method</term><term>Female</term><term>Genetic Therapy (methods)</term><term>Genetic Vectors (therapeutic use)</term><term>Humans</term><term>Male</term><term>Middle Aged</term><term>Neurturin (genetics)</term><term>Parkinson Disease (metabolism)</term><term>Parkinson Disease (physiopathology)</term><term>Parkinson Disease (therapy)</term><term>Putamen (metabolism)</term><term>Putamen (physiopathology)</term><term>Substantia Nigra (metabolism)</term><term>Substantia Nigra (physiopathology)</term><term>Treatment Outcome</term></keywords><keywords scheme="MESH" type="chemical" qualifier="genetics" xml:lang="en"><term>Neurturin</term></keywords><keywords scheme="MESH" qualifier="metabolism" xml:lang="en"><term>Parkinson Disease</term><term>Putamen</term><term>Substantia Nigra</term></keywords><keywords scheme="MESH" qualifier="methods" xml:lang="en"><term>Genetic Therapy</term></keywords><keywords scheme="MESH" qualifier="physiopathology" xml:lang="en"><term>Parkinson Disease</term><term>Putamen</term><term>Substantia Nigra</term></keywords><keywords scheme="MESH" qualifier="therapeutic use" xml:lang="en"><term>Genetic Vectors</term></keywords><keywords scheme="MESH" qualifier="therapy" xml:lang="en"><term>Parkinson Disease</term></keywords><keywords scheme="MESH" xml:lang="en"><term>Aged</term><term>Axonal Transport</term><term>Dependovirus</term><term>Double-Blind Method</term><term>Female</term><term>Humans</term><term>Male</term><term>Middle Aged</term><term>Treatment Outcome</term></keywords></textClass></profileDesc></teiHeader><front><div type="abstract" xml:lang="en">A 12-month double-blind sham-surgery-controlled trial assessing adeno-associated virus type 2 (AAV2)-neurturin injected into the putamen bilaterally failed to meet its primary endpoint, but showed positive results for the primary endpoint in the subgroup of subjects followed for 18 months and for several secondary endpoints. Analysis of postmortem tissue suggested impaired axonal transport of neurturin from putamen to substantia nigra. In the present study, we tested the safety and efficacy of AAV2-neurturin delivered to putamen and substantia nigra.</div></front></TEI><affiliations><list><country><li>Canada</li><li>États-Unis</li></country><region><li>Alabama</li><li>Californie</li><li>Caroline du Nord</li><li>Géorgie (États-Unis)</li><li>Illinois</li><li>Massachusetts</li><li>Ontario</li><li>Pennsylvanie</li><li>Texas</li><li>État de New York</li></region><settlement><li>Houston</li><li>Toronto</li></settlement><orgName><li>Baylor College of Medicine</li><li>Université de Toronto</li></orgName></list><tree><country name="États-Unis"><region name="État de New York"><name sortKey="Warren Olanow, C" sort="Warren Olanow, C" uniqKey="Warren Olanow C" first="C" last="Warren Olanow">C. Warren Olanow</name></region><name sortKey="Alterman, Ron" sort="Alterman, Ron" uniqKey="Alterman R" first="Ron" last="Alterman">Ron Alterman</name><name sortKey="Baltuch, Gordon" sort="Baltuch, Gordon" uniqKey="Baltuch G" first="Gordon" last="Baltuch">Gordon Baltuch</name><name sortKey="Bartus, Raymond T" sort="Bartus, Raymond T" uniqKey="Bartus R" first="Raymond T" last="Bartus">Raymond T. Bartus</name><name sortKey="Baumann, Tiffany L" sort="Baumann, Tiffany L" uniqKey="Baumann T" first="Tiffany L" last="Baumann">Tiffany L. Baumann</name><name sortKey="Boulis, Nicholas" sort="Boulis, Nicholas" uniqKey="Boulis N" first="Nicholas" last="Boulis">Nicholas Boulis</name><name sortKey="Factor, Stewart" sort="Factor, Stewart" uniqKey="Factor S" first="Stewart" last="Factor">Stewart Factor</name><name sortKey="Goetz, Christopher G" sort="Goetz, Christopher G" uniqKey="Goetz C" first="Christopher G" last="Goetz">Christopher G. Goetz</name><name sortKey="Guthrie, Barton" sort="Guthrie, Barton" uniqKey="Guthrie B" first="Barton" last="Guthrie">Barton Guthrie</name><name sortKey="Henderson, Jaimie M" sort="Henderson, Jaimie M" uniqKey="Henderson J" first="Jaimie M" last="Henderson">Jaimie M. Henderson</name><name sortKey="Herzog, Christopher" sort="Herzog, Christopher" uniqKey="Herzog C" first="Christopher" last="Herzog">Christopher Herzog</name><name sortKey="Jankovic, Joseph" sort="Jankovic, Joseph" uniqKey="Jankovic J" first="Joseph" last="Jankovic">Joseph Jankovic</name><name sortKey="Kordower, Jeffrey H" sort="Kordower, Jeffrey H" uniqKey="Kordower J" first="Jeffrey H" last="Kordower">Jeffrey H. Kordower</name><name sortKey="Larson, Paul" sort="Larson, Paul" uniqKey="Larson P" first="Paul" last="Larson">Paul Larson</name><name sortKey="Marks, William" sort="Marks, William" uniqKey="Marks W" first="William" last="Marks">William Marks</name><name sortKey="Poston, Kathleen" sort="Poston, Kathleen" uniqKey="Poston K" first="Kathleen" last="Poston">Kathleen Poston</name><name sortKey="Simpson, Richard" sort="Simpson, Richard" uniqKey="Simpson R" first="Richard" last="Simpson">Richard Simpson</name><name sortKey="Stacy, Mark" sort="Stacy, Mark" uniqKey="Stacy M" first="Mark" last="Stacy">Mark Stacy</name><name sortKey="Starr, Phillip A" sort="Starr, Phillip A" uniqKey="Starr P" first="Phillip A" last="Starr">Phillip A. Starr</name><name sortKey="Stern, Matthew" sort="Stern, Matthew" uniqKey="Stern M" first="Matthew" last="Stern">Matthew Stern</name><name sortKey="Turner, Dennis A" sort="Turner, Dennis A" uniqKey="Turner D" first="Dennis A" last="Turner">Dennis A. Turner</name><name sortKey="Watts, Ray" sort="Watts, Ray" uniqKey="Watts R" first="Ray" last="Watts">Ray Watts</name></country><country name="Canada"><region name="Ontario"><name sortKey="Lozano, Andres M" sort="Lozano, Andres M" uniqKey="Lozano A" first="Andres M" last="Lozano">Andres M. Lozano</name></region><name sortKey="Lang, Anthony E" sort="Lang, Anthony E" uniqKey="Lang A" first="Anthony E" last="Lang">Anthony E. Lang</name></country></tree></affiliations></record>Pour manipuler ce document sous Unix (Dilib)
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