Compounds Target Disease Variants
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potential new therapeutic strategiesPhys Org
•Scientists create vast library of compounds to target disease proteins
90% Informative
Researchers at the Broad Institute of MIT and Harvard , Massachusetts General Hospital , and Harvard University build a diverse collection of molecular compounds that can be mined for those that target disease-related genetic variants in new ways.
The library includes more than 3 million compounds designed to bring two proteins together and use one as a shield to stabilize the other and reverse its effects.
The approach could potentially be used to recruit proteins with other functional effects or target disease risk factors in only certain cell types or tissues.
The CIP-DEL library stabilizes the protein, protects it from caspase-3 cleavage and reverses the impairment of autophagy and other cellular processes.
The researchers aim to next develop a version of the compound that works in animal models so they can further explore therapeutic opportunities.
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