Products and methods for gaucher disease therapy
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United States of America Patent
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Feb 24, 2004
Grant Date -
N/A
app pub date -
Jun 2, 2000
filing date -
Jun 2, 1999
priority date (Note) -
Expired
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Abstract
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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