20250152747. Methods Treating Gauche (Genzyme)
METHODS OF TREATING GAUCHER DISEASE AND GBA-PARKINSONâS DISEASE
Abstract: provided herein are expression cassettes for expressing a transgene in a cell, wherein the transgene encodes a gcase polypeptide. also provided are methods to treat gaucher disease or gba-pd. further provided herein are vectors (e.g., raav vectors), viral particles, pharmaceutical compositions, and kits for expressing an gcase polypeptide in an individual in need thereof.
Inventor(s): Swathi AYLOO, Shyam RAMACHANDRAN
CPC Classification: A61K48/0066 (Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy)
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