Systemic AAV9 gene therapy using the synapsin I promoter rescues a mouse model of neuronopathic Gaucher disease but with limited cross-correction potential to astrocytes

Gaucher disease is caused by mutations in the GBA gene, which encodes for the lysosomal enzyme β-glucocerebrosidase (GCase), resulting in the accumulation of storage material in visceral organs and in some cases the brain of affected patients. While there is a commercially available treatment for th...

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Detalles Bibliográficos
Autores principales: Massaro, Giulia, Hughes, Michael P, Whaler, Sammie M, Wallom, Kerri-Lee, Priestman, David A, Platt, Frances M, Waddington, Simon N, Rahim, Ahad A
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2020
Materias:
General Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7390934/
https://www.ncbi.nlm.nih.gov/pubmed/31919491
http://dx.doi.org/10.1093/hmg/ddz317

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7390934/
https://www.ncbi.nlm.nih.gov/pubmed/31919491
http://dx.doi.org/10.1093/hmg/ddz317

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