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TREATING LYSOSOMAL STORAGE DISEASE
专利权人:
UNIVERSITY OF ROCHESTER
发明人:
Mark Noble,Christopher Folts,Nicole Scotts
申请号:
US14777100
公开号:
US20160022775A1
申请日:
2014.03.14
申请国别(地区):
US
年份:
2016
代理人:
摘要:
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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