Altif Laboratories

Tokyo Metropolitan Institute of Medical Science

Tokyo Metropolitan Organization for Medical Research

Seiichi Aikawa

Hitoshi Sakuraba

Youichi Tajima

Fumiko Aikawa

Mai Ito

Ikuo Kawashima

The present invention provides a pharmaceutical composition comprising a protein having &#x3b1;-galactosidase activity for treating Fabry disease, which causes no allergic side effect, which is highly stable in blood (plasma) and which can readily be taken up by a cell of an affected organ. The pharmaceutical composition for treating Fabry disease of the invention comprises, for example, a protein which acquires an &#x3b1;-galactosidase activity through alteration of the structure of the active site of wild-type human &#x3b1;-N-acetylgalactosaminidase.

Polynucleotides encoding proteins having acquired &#x3b1;-galactosidase activity

The present invention provides, as an enzyme which can be used for enzyme replacement therapy for Fabry disease, a protein having &#x3b1;-galactosidase activity, which shows no allergic adverse side effect, shows a high stability in blood, and can be easily incorporated into a cell of an affected organ. The protein of the present invention is a protein which has acquired &#x3b1;-galactosidase activity by changing the structure of the active site of wild-type human &#x3b1;-N-acetylgalactosaminidase.

Highly functional enzyme having alpha-galactosidase activity

Highly functional enzyme having &#x3b1;-galactosidase activity

Proteins having acquired A-galactosidase activity

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Tokyo, Japan

Seiichi Aikawa