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Efficacy of a Bicistronic Vector for Correction of Sandhoff Disease in a Mouse Model

G(M2) gangliosidoses are a family of severe neurodegenerative disorders resulting from a deficiency in the β-hexosaminidase A enzyme. These disorders include Tay-Sachs disease and Sandhoff disease, caused by mutations in the HEXA gene and HEXB gene, respectively. The HEXA and HEXB genes are required...

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Detalles Bibliográficos
Autores principales:	Woodley, Evan, Osmon, Karlaina J.L., Thompson, Patrick, Richmond, Christopher, Chen, Zhilin, Gray, Steven J., Walia, Jagdeep S.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2018
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6279944/ https://www.ncbi.nlm.nih.gov/pubmed/30534578 http://dx.doi.org/10.1016/j.omtm.2018.10.011

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