Gene therapy for lysosomal storage diseases
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United States of America Patent
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Aug 5, 2014
Grant Date -
Jul 8, 2010
app pub date -
Dec 4, 2009
filing date -
Jun 6, 2007
priority date (Note) -
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Abstract
This disclosure provides methods and compositions for treating lysosomal storage diseases in a subject. In one aspect of the invention, a transgene product is delivered to a subject by administering a recombinant neurotrophic viral vector containing the transgene to the brain. The viral vector delivers the transgene to a region of the brain which is susceptible to infection by the virus and which expresses the encoded recombinant viral gene product. Also provided are compositions for delivery of a transgene product to a subject by administering a recombinant neurotrophic viral vector containing the transgene to the subject's brain. The transgene product may be any that is deficient in a lysosomal storage disease.
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