Article

Promoter or enhancer activation by CRISPRa rescues haploinsufficiency caused obesity

2017-05-22

Abstract excerpt

Haploinsufficiency, having only one functional copy of a gene, leads to a wide range of human disease and has been associated with over 300 genes. Here, we tested whether CRISPR activation (CRISPRa) could rescue a haploinsufficient disease in vivo . Haploinsufficiency of Sim1 , a transcription factor involved in the leptin pathway, results in severe obesity in humans and mice. CRISPRa targeting of either the Si...

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Literature Corpus work: dc785eca-10d9-56bc-ab29-83317855afaf
DOI: 10.1101/140426

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