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RAAV Vectors for the Treatment of GM1 and GM2 Gangliosidosis
RAAV Vectors for the Treatment of GM1 and GM2 Gangliosidosis
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机译:rav载体治疗GM1和GM2神经节病病
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摘要
Aspects of the disclosure relate to compositions and methods for the treatment of lysosomal storage disorders, such as GM1 gangliosidosis, Tay Sachs disease, and Sandhoff disease. In some embodiments, the compositions comprise viral vectors encoding beta-galactosidase. In some embodiments, the compositions comprise viral vectors encoding beta-hexosaminidase subunits (e.g. HEXA, HEXB, or combinations thereof).
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