Article

First-in-human AAV Gene Therapy for Tay-Sachs Disease

2021-02-18

Abstract excerpt

<title>Abstract</title> <p>Tay-Sachs Disease (TSD) is an inherited neurological disorder caused by deficiency of hexosaminidase A (HexA). Preclinical work demonstrated safety and efficacy of CNS gene therapy using AAVrh8-HEXA/HEXB. Here we describe an expanded access trial in two patients with infantile TSD (IND 18225). Case TSD-001 demonstrated neurodevelopmental regression by 8 months of age and severe seizures...

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Literature Corpus work: 5b256d37-ff3a-5ecb-9f33-f98c9c5134f6
DOI: 10.21203/rs.3.rs-195847/v1

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