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Gene Therapy for Parkinson’s Disease Associated with GBA1 Mutations

Human genetic studies as well as studies in animal models indicate that lysosomal dysfunction plays a key role in the pathogenesis of Parkinson’s disease. Among the lysosomal genes involved, GBA1 has the largest impact on Parkinson’s disease risk. Deficiency in the GBA1 encoded enzyme glucocerebrosi...

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Detalles Bibliográficos
Autores principales: Abeliovich, Asa, Hefti, Franz, Sevigny, Jeffrey
Formato: Online Artículo Texto
Lenguaje:English
Publicado: IOS Press 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8543272/
https://www.ncbi.nlm.nih.gov/pubmed/34151863
http://dx.doi.org/10.3233/JPD-212739