Development of gene therapy for lysosomal disease with central nervous system based on preclinical and clinical trials


Ontology type: schema:MonetaryGrant     


Grant Info

YEARS

2017-2018

FUNDING AMOUNT

78000000.0 JPY

ABSTRACT

Preparation of modified human Hex β-chain gene-loaded AAV vector (GMP grade) expressing modified Hex B for treatment of Tay-Sachs disease and Sandhoff's disease, and implementation of non-clinical study of gene therapy by intrathecal administration

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