Gene delivery of AAV2-neurturin for Parkinson's disease: a double-blind, randomised, controlled trial.
Identifieur interne : 000520 ( Main/Merge ); précédent : 000519; suivant : 000521Gene delivery of AAV2-neurturin for Parkinson's disease: a double-blind, randomised, controlled trial.
Auteurs : William. Marks [États-Unis] ; Raymond. Bartus ; Joao Siffert ; Charles. Davis ; Andres Lozano ; Nicholas Boulis ; Jerrold Vitek ; Mark Stacy ; Dennis Turner ; Leonard Verhagen ; Roy Bakay ; Raymond Watts ; Barton Guthrie ; Joseph Jankovic [États-Unis] ; Richard Simpson ; Michele Tagliati ; Ron Alterman ; Matthew Stern ; Gordon Baltuch ; Philip. Starr ; Paul. Larson ; Jill. Ostrem ; John Nutt ; Karl Kieburtz ; Jeffrey. Kordower ; C OlanowSource :
- The Lancet. Neurology ; 2010.
English descriptors
- KwdEn :
- Adult, Aged, Analysis of Variance, Dependovirus (genetics), Dependovirus (physiology), Double-Blind Method, Female, Follow-Up Studies, Genetic Therapy (methods), Humans, Male, Middle Aged, Motor Activity (physiology), Neurturin (genetics), Neurturin (metabolism), Neurturin (therapeutic use), Parkinson Disease (genetics), Parkinson Disease (physiopathology), Parkinson Disease (therapy), Putamen (metabolism), Putamen (physiology), Severity of Illness Index, Time Factors, Treatment Outcome.
- MESH :
- chemical , genetics : Neurturin.
- genetics : Dependovirus, Parkinson Disease.
- chemical , metabolism : Neurturin, Putamen.
- methods : Genetic Therapy.
- physiology : Dependovirus, Motor Activity, Putamen.
- physiopathology : Parkinson Disease.
- chemical , therapeutic use : Neurturin.
- therapy : Parkinson Disease.
- Adult, Aged, Analysis of Variance, Double-Blind Method, Female, Follow-Up Studies, Humans, Male, Middle Aged, Severity of Illness Index, Time Factors, Treatment Outcome.
Abstract
In an open-label phase 1 trial, gene delivery of the trophic factor neurturin via an adeno-associated type-2 vector (AAV2) was well tolerated and seemed to improve motor function in patients with advanced Parkinson's disease. We aimed to assess the safety and efficacy of AAV2-neurturin in a double-blind, phase 2 randomised trial.
DOI: 10.1016/S1474-4422(10)70254-4
PubMed: 20970382
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pubmed:20970382Le document en format XML
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Bartus</name></author><author><name sortKey="Siffert, Joao" sort="Siffert, Joao" uniqKey="Siffert J" first="Joao" last="Siffert">Joao Siffert</name></author><author><name sortKey="Davis, Charles S" sort="Davis, Charles S" uniqKey="Davis C" first="Charles" last="Davis">Charles. Davis</name></author><author><name sortKey="Lozano, Andres" sort="Lozano, Andres" uniqKey="Lozano A" first="Andres" last="Lozano">Andres Lozano</name></author><author><name sortKey="Boulis, Nicholas" sort="Boulis, Nicholas" uniqKey="Boulis N" first="Nicholas" last="Boulis">Nicholas Boulis</name></author><author><name sortKey="Vitek, Jerrold" sort="Vitek, Jerrold" uniqKey="Vitek J" first="Jerrold" last="Vitek">Jerrold Vitek</name></author><author><name sortKey="Stacy, Mark" sort="Stacy, Mark" uniqKey="Stacy M" first="Mark" last="Stacy">Mark Stacy</name></author><author><name sortKey="Turner, Dennis" sort="Turner, Dennis" uniqKey="Turner D" first="Dennis" last="Turner">Dennis Turner</name></author><author><name sortKey="Verhagen, Leonard" sort="Verhagen, Leonard" uniqKey="Verhagen L" first="Leonard" last="Verhagen">Leonard Verhagen</name></author><author><name sortKey="Bakay, Roy" sort="Bakay, Roy" uniqKey="Bakay R" first="Roy" last="Bakay">Roy Bakay</name></author><author><name sortKey="Watts, Raymond" sort="Watts, Raymond" uniqKey="Watts R" first="Raymond" last="Watts">Raymond Watts</name></author><author><name sortKey="Guthrie, Barton" sort="Guthrie, Barton" uniqKey="Guthrie B" first="Barton" last="Guthrie">Barton Guthrie</name></author><author><name sortKey="Jankovic, Joseph" sort="Jankovic, Joseph" uniqKey="Jankovic J" first="Joseph" last="Jankovic">Joseph Jankovic</name><affiliation><country>États-Unis</country><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></author><author><name sortKey="Tagliati, Michele" sort="Tagliati, Michele" uniqKey="Tagliati M" first="Michele" last="Tagliati">Michele Tagliati</name></author><author><name sortKey="Alterman, Ron" sort="Alterman, Ron" uniqKey="Alterman R" first="Ron" last="Alterman">Ron Alterman</name></author><author><name sortKey="Stern, Matthew" sort="Stern, Matthew" uniqKey="Stern M" first="Matthew" last="Stern">Matthew Stern</name></author><author><name sortKey="Baltuch, Gordon" sort="Baltuch, Gordon" uniqKey="Baltuch G" first="Gordon" last="Baltuch">Gordon Baltuch</name></author><author><name sortKey="Starr, Philip A" sort="Starr, Philip A" uniqKey="Starr P" first="Philip" last="Starr">Philip. 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Marks</name><affiliation wicri:level="2"><nlm:affiliation>Department of Neurology, University of California San Francisco, San Francisco, CA, USA.</nlm:affiliation><country xml:lang="fr">États-Unis</country><wicri:regionArea>Department of Neurology, University of California San Francisco, San Francisco, CA</wicri:regionArea><placeName><region type="state">Californie</region></placeName></affiliation></author><author><name sortKey="Bartus, Raymond T" sort="Bartus, Raymond T" uniqKey="Bartus R" first="Raymond" last="Bartus">Raymond. Bartus</name></author><author><name sortKey="Siffert, Joao" sort="Siffert, Joao" uniqKey="Siffert J" first="Joao" last="Siffert">Joao Siffert</name></author><author><name sortKey="Davis, Charles S" sort="Davis, Charles S" uniqKey="Davis C" first="Charles" last="Davis">Charles. Davis</name></author><author><name sortKey="Lozano, Andres" sort="Lozano, Andres" uniqKey="Lozano A" first="Andres" last="Lozano">Andres Lozano</name></author><author><name sortKey="Boulis, Nicholas" sort="Boulis, Nicholas" uniqKey="Boulis N" first="Nicholas" last="Boulis">Nicholas Boulis</name></author><author><name sortKey="Vitek, Jerrold" sort="Vitek, Jerrold" uniqKey="Vitek J" first="Jerrold" last="Vitek">Jerrold Vitek</name></author><author><name sortKey="Stacy, Mark" sort="Stacy, Mark" uniqKey="Stacy M" first="Mark" last="Stacy">Mark Stacy</name></author><author><name sortKey="Turner, Dennis" sort="Turner, Dennis" uniqKey="Turner D" first="Dennis" last="Turner">Dennis Turner</name></author><author><name sortKey="Verhagen, Leonard" sort="Verhagen, Leonard" uniqKey="Verhagen L" first="Leonard" last="Verhagen">Leonard Verhagen</name></author><author><name sortKey="Bakay, Roy" sort="Bakay, Roy" uniqKey="Bakay R" first="Roy" last="Bakay">Roy Bakay</name></author><author><name sortKey="Watts, Raymond" sort="Watts, Raymond" uniqKey="Watts R" first="Raymond" last="Watts">Raymond Watts</name></author><author><name sortKey="Guthrie, Barton" sort="Guthrie, Barton" uniqKey="Guthrie B" first="Barton" last="Guthrie">Barton Guthrie</name></author><author><name sortKey="Jankovic, Joseph" sort="Jankovic, Joseph" uniqKey="Jankovic J" first="Joseph" last="Jankovic">Joseph Jankovic</name><affiliation><country>États-Unis</country><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></author><author><name sortKey="Tagliati, Michele" sort="Tagliati, Michele" uniqKey="Tagliati M" first="Michele" last="Tagliati">Michele Tagliati</name></author><author><name sortKey="Alterman, Ron" sort="Alterman, Ron" uniqKey="Alterman R" first="Ron" last="Alterman">Ron Alterman</name></author><author><name sortKey="Stern, Matthew" sort="Stern, Matthew" uniqKey="Stern M" first="Matthew" last="Stern">Matthew Stern</name></author><author><name sortKey="Baltuch, Gordon" sort="Baltuch, Gordon" uniqKey="Baltuch G" first="Gordon" last="Baltuch">Gordon Baltuch</name></author><author><name sortKey="Starr, Philip A" sort="Starr, Philip A" uniqKey="Starr P" first="Philip" last="Starr">Philip. 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Neurology</title><idno type="e-ISSN">1474-4465</idno><imprint><date when="2010" type="published">2010</date></imprint></series></biblStruct></sourceDesc></fileDesc><profileDesc><textClass><keywords scheme="KwdEn" xml:lang="en"><term>Adult</term><term>Aged</term><term>Analysis of Variance</term><term>Dependovirus (genetics)</term><term>Dependovirus (physiology)</term><term>Double-Blind Method</term><term>Female</term><term>Follow-Up Studies</term><term>Genetic Therapy (methods)</term><term>Humans</term><term>Male</term><term>Middle Aged</term><term>Motor Activity (physiology)</term><term>Neurturin (genetics)</term><term>Neurturin (metabolism)</term><term>Neurturin (therapeutic use)</term><term>Parkinson Disease (genetics)</term><term>Parkinson Disease (physiopathology)</term><term>Parkinson Disease (therapy)</term><term>Putamen (metabolism)</term><term>Putamen (physiology)</term><term>Severity of Illness Index</term><term>Time Factors</term><term>Treatment Outcome</term></keywords><keywords scheme="MESH" type="chemical" qualifier="genetics" xml:lang="en"><term>Neurturin</term></keywords><keywords scheme="MESH" qualifier="genetics" xml:lang="en"><term>Dependovirus</term><term>Parkinson Disease</term></keywords><keywords scheme="MESH" type="chemical" qualifier="metabolism" xml:lang="en"><term>Neurturin</term><term>Putamen</term></keywords><keywords scheme="MESH" qualifier="methods" xml:lang="en"><term>Genetic Therapy</term></keywords><keywords scheme="MESH" qualifier="physiology" xml:lang="en"><term>Dependovirus</term><term>Motor Activity</term><term>Putamen</term></keywords><keywords scheme="MESH" qualifier="physiopathology" xml:lang="en"><term>Parkinson Disease</term></keywords><keywords scheme="MESH" type="chemical" qualifier="therapeutic use" xml:lang="en"><term>Neurturin</term></keywords><keywords scheme="MESH" qualifier="therapy" xml:lang="en"><term>Parkinson Disease</term></keywords><keywords scheme="MESH" xml:lang="en"><term>Adult</term><term>Aged</term><term>Analysis of Variance</term><term>Double-Blind Method</term><term>Female</term><term>Follow-Up Studies</term><term>Humans</term><term>Male</term><term>Middle Aged</term><term>Severity of Illness Index</term><term>Time Factors</term><term>Treatment Outcome</term></keywords></textClass></profileDesc></teiHeader><front><div type="abstract" xml:lang="en">In an open-label phase 1 trial, gene delivery of the trophic factor neurturin via an adeno-associated type-2 vector (AAV2) was well tolerated and seemed to improve motor function in patients with advanced Parkinson's disease. We aimed to assess the safety and efficacy of AAV2-neurturin in a double-blind, phase 2 randomised trial.</div></front></TEI></record>Pour manipuler ce document sous Unix (Dilib)
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