Therapy for .alpha.-galactosidase a deficiency

Details Therapy for .alpha.-galactosidase a deficiency Therapy for .alpha.-galactosidase a deficiency

C - Chemistry, Metallurgy

12

N

C12N 15/56 (2006.01) A61K 38/47 (2006.01) A61K 48/00 (2006.01) C07K 14/61 (2006.01) C12N 5/10 (2006.01) C12N 9/40 (2006.01) C12N 15/62 (2006.01) C12P 21/02 (2006.01) A61K 38/00 (2006.01)

Patent

CA 2265464

A therapeutic method whereby an individual suspected of having an .alpha.- galactosidase A deficiency, such as Fabry disease, is treated either with (I) human cells that have been genetically modified to overexpress and secrete human .alpha.-gal A, or (2) purified human .alpha.-gal A obtained from cultured, genetically modified human cells.

L'invention concerne une méthode thérapeutique par laquelle un sujet soupçonné de présenter une carence en alpha -galactosidase A, telle que la maladie de Fabry, est traité soit avec (1) des cellules humaines ayant été génétiquement modifiées pour surexprimer et sécréter de l' alpha -galactosidase A humaine, soit avec (2) de l' alpha -galactosidase A humaine purifiée obtenue à partir de cellules humaines génétiquement modifiées mises en culture.

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