Gene delivery of AAV2-neurturin for Parkinson's disease: a double-blind, randomised, controlled trial.
Identifieur interne : 000308 ( Ncbi/Checkpoint ); précédent : 000307; suivant : 000309Gene 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
Affiliations:
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pubmed:20970382Le document en format XML
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<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>
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