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Article

Therapeutic Response in Feline Sandhoff Disease Despite Immunity to Intracranial Gene Therapy

21 May 2013

Abstract excerpt

Salutary responses to adeno-associated viral (AAV) gene therapy have been reported in the mouse model of Sandhoff disease (SD), a neurodegenerative lysosomal storage disease caused by deficiency of β-N-acetylhexosaminidase (Hex). While untreated mice reach the humane endpoint by 4.1 months of age, mice treated by a single intracranial injection of vectors expressing human hexosaminidase may live a normal life...

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PMID: 23689599
DOI: 10.1038/mt.2013.86

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