Products and methods for gaucher disease therapy

Details Products and methods for gaucher disease therapy Products and methods for gaucher disease therapy

C - Chemistry, Metallurgy

12

N

C12N 15/12 (2006.01) A61K 9/127 (2006.01) A61K 31/70 (2006.01) A61K 48/00 (2006.01) C12N 5/10 (2006.01) C12N 9/24 (2006.01) C12N 15/63 (2006.01)

Patent

CA 2272055

The invention relates to products and methods for medical treatment of Gaucher disease and, in particular, an improved Gcc DNA for insertion into any applicable expression vector for gene therapy treatment. The invention includes an isolated Gcc DNA molecule, wherein nucleic acid molecules have been modified at cryptic splice sites to prevent or decrease splicing of mRNA produced from the DNA molecule, while preserving the ability of the DNA to express functional Gcc polypeptides.

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