Novel highly functional enzyme having modified...

Details Novel highly functional enzyme having modified... Novel highly functional enzyme having modified...

C - Chemistry, Metallurgy

12

N

C12N 15/56 (2006.01) A61K 31/7088 (2006.01) A61K 38/47 (2006.01) A61K 48/00 (2006.01) A61P 3/00 (2006.01) C12N 9/40 (2006.01) C12N 15/09 (2006.01)

Patent

CA 2626092

Disclosed is a protein having an .alpha.-galactosidase activity, which shows no allergic adverse side effect, shows a high stability in the blood and can be taken into a cell of an affected organ, and therefore is suitable as an enzyme for enzyme replacement therapy on Fabry disease, The protein acquires an .alpha.-galactosidase activity by modifying the structure of the active site of a wild-type human .alpha.-N-acetylgalactosaminidase.

L'invention concerne une protéine ayant une activité d'.alpha.-galactosidase, laquelle ne présente pas d'effet secondaire négatif de type allergique, présente une stabilité élevée dans le sang et peut être absorbée dans une cellule d'un organe affecté et par conséquent convient comme enzyme pour une enzymothérapie substitutive sur la maladie de Fabry. La protéine acquiert une activité d'.alpha.-galactosidase en modifiant la structure du site actif d'une .alpha.-N-acétylgalactosaminidase humaine de type sauvage.

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