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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...
Autores principales: | Abeliovich, Asa, Hefti, Franz, Sevigny, Jeffrey |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
IOS Press
2021
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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 |
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