METHOD OF TREATING GENETIC DISORDERS
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United States of America Patent
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Issued Date -
May 19, 2011
app pub date -
Mar 30, 2009
filing date -
Apr 2, 2008
priority date (Note) -
Abandoned
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Abstract
A method for the treatment of diseases associated with mutations in MYO7A or CEP290 genes, especially the Usher Syndrome type IB and Leber congenital amaurosis, by administering to a subject in need thereof an adeno-associated viral vector encoding a MYO7A or a CEP290 protein; genetic constructs and adeno-associated viral vectors for use in this method.
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