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Therapeutic Strategies For Tay-Sachs Disease

5 Jul 2022

Abstract excerpt

Tay-Sachs disease (TSD) is an autosomal recessive disease that features progressive neurodegenerative presentations. It affects one in 100,000 live births. Currently, there is no approved therapy or cure. This review summarizes multiple drug development strategies for TSD, including enzyme replacement therapy, pharmaceutical chaperone therapy, substrate reduction therapy, gene therapy, and hematopoietic stem cell...

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PMID: 35865957
DOI: 10.3389/fphar.2022.906647

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