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SYNTHETIC MBNL PROTEINS FOR TREATMENT OF REPEAT EXPANSION DISEASE
专利权人:
发明人:
John A. Berglund,Melissa Hale
申请号:
US16308098
公开号:
US20190177380A1
申请日:
2017.08.06
申请国别(地区):
US
年份:
2019
代理人:
摘要:
Provided herein are compositions and methods related to treating repeat expansion diseases that feature the sequestration of Muscleblind-like (MBNL) proteins by the toxic repeat RNA transcripts within distinct nuclear foci that are expressed from the expanded repeat tracts. Certain compositions of synthetic MBLN proteins can be used to displace endogenous MBNL from the toxic RNAs or replace endogenous MBNL within the cell for normal function.
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