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   Articles with "corrects haploinsufficient" as a keyword



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CRISPRa corrects haploinsufficient obesity

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recommendations! Published in 2019 at "Science"

DOI: 10.1126/science.363.6424.241-l

Abstract: Gene Therapy Loss-of-function mutation in one gene copy, termed haploinsufficiency, can lead to insufficient protein levels and result in human disease. Matharu et al. tested whether a CRISPR-based activation system (CRISPRa) could rescue a haploinsufficient… read more here.

Keywords: science; haploinsufficient obesity; corrects haploinsufficient; obesity ... See more keywords