CRISPR-mediated activation of a promoter or enhancer rescues obesity caused by haploinsufficiency

Abstract excerpt

CRISPRa corrects haploinsufficient obesity 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 phenotype by increasing the gene expression levels of the existing normal copy (see the Perspective by Montefiori and Nobrega)....

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