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beta-Hexosaminidase alpha Chain
beta-Hexosaminidase beta Chain
Adeno-Associated Virus Gene Therapy in a Sheep Model of Tay-Sachs Disease.
Biomarkers for disease progression and AAV therapeutic efficacy in feline Sandhoff disease.
Mucopolysaccharidosis-like phenotype in feline Sandhoff disease and partial correction after AAV gene therapy.
Therapeutic response in feline sandhoff disease despite immunity to intracranial gene therapy.
Widespread correction of central nervous system disease after intracranial gene therapy in a feline model of Sandhoff disease.
Pronounced Therapeutic Benefit of a Single Bidirectional AAV Vector Administered Systemically in Sandhoff Mice.
Therapeutic benefit after intracranial gene therapy delivered during the symptomatic stage in a feline model of Sandhoff disease.
beta N Acetylhexosaminidases