Polynucleotides encoding proteins having acquired α-galactosidase activity
AssigneesTokyo Metropolitan Institute of Medical Science, Altif Laboratories
InventorsHitoshi Sakuraba, Youichi Tajima, Ikuo Kawashima, Seiichi Aikawa, Fumiko Aikawa
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Abstract
The present invention provides a pharmaceutical composition comprising a protein having α-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 α-galactosidase activity through alteration of the structure of the active site of wild-type human α-N-acetylgalactosaminidase.
Claims7
An isolated nucleic acid molecule comprising a nucleotide sequence having at least 95% identity to SEQ ID NO:
- 5 wherein
- the nucleotides 604-606 of SEQ ID NO:5 are substituted by nucleotides encoding an amino acid other than serine,
- the nucleotides 613-615 of SEQ ID NO:5 are substituted by nucleotides encoding an amino acid other than alanine, and
- the nucleotide sequence encodes a protein having α-galactosidase activity.
Dependencies are inferred from the claim text.