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Adeno-Associated Virus Gene Therapy in a Sheep Model of Tay–Sachs Disease

10 Nov 2017

Abstract excerpt

Tay–Sachs disease (TSD) is a fatal neurodegenerative disorder caused by a deficiency of the enzyme hexosaminidase A (HexA). TSD also occurs in sheep, the only experimental model of TSD that has clinical signs of disease. The natural history of sheep TSD was characterized using serial neurological evaluations, 7 Tesla magnetic resonance imaging, echocardiograms, electrodiagnostics, and cerebrospinal fluid...

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PMID: 28922945
DOI: 10.1089/hum.2017.163

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