THERAPY FOR α-GALACTOSIDASE A DEFICIENCY

摘要

PROBLEM TO BE SOLVED: To provide human cells for implantation genetically modified to overexpress and secrete human α-gal A, and purified human α-gal A, for treating an individual suspected of having an α-galactosidase A deficiency, such as Fabry disease.SOLUTION: Compositions for therapy are provided for treating with either (1) human cells genetically modified to overexpress and secrete human α-gal A, or (2) purified human α-gal A obtained from cultured, genetically modified human cells.