TREATING LYSOSOMAL STORAGE DISEASE
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United States of America Patent
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Jan 28, 2016
app pub date -
Mar 14, 2014
filing date -
Mar 15, 2013
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Abstract
Provided herein are compositions, kits and methods related to the treatment of one or more lysosomal storage diseases (LSDs) in a subject and methods of identifying agents that are useful for the treatment of one or more LSDs. The compositions, kits, and methods are based on the novel discovery that agents effective for treating LSD must have two characteristics: they must inhibit cell death and reduce suppression of cell division caused by toxic substances that accumulate in LSD. Exemplary agents include polypeptides (e.g., IGF-1, VEGF) and small molecules (e.g., chlorotrianisene, clofoctal, colforsin, and tulobuterol).
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