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Immunomodulatory Effects of Intracranial Gene Therapy in Feline Sandhoff Disease
Bradbury, Allison (2014-05-02)
Sandhoff disease (SD) is a lysosomal storage disorder characterized by absence of hydrolytic enzyme β-N-acetylhexosaminidase (Hex), which results in storage of GM2 ganglioside in neurons and unremitting neurodegeneration. ...