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Small-molecule enhancers of CRISPR-induced homology-directed repair in gene therapy: A medicinal chemist's perspective

期刊

DRUG DISCOVERY TODAY
卷 27, 期 9, 页码 2510-2525

出版社

ELSEVIER SCI LTD
DOI: 10.1016/j.drudis.2022.06.006

关键词

Small molecules; CRISPR; Genome editing; Homology-directed repair; DNA repair; Gene therapy

资金

  1. A*STAR under its A*STAR Graduate Scholarship programme
  2. Ministry of Education's Academic Research Fund (MOE AcRF Tier 1) [A0004633-00-00]

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Using small molecules to increase the efficiency of HDR and decrease NHEJ to improve the efficiency of precise knock-in genome editing.
CRISPR technologies are increasingly being investigated and utilized for the treatment of human genetic diseases via genome editing. CRISPR-Cas9 first generates a targeted DNA double-stranded break, and a functional gene can then be introduced to replace the defective copy in a precise manner by templated repair via the homology -directed repair (HDR) pathway. However, this is challeng-ing owing to the relatively low efficiency of the HDR pathway compared with a rival random repair pathway known as non-homologous end joining (NHEJ). Small molecules can be employed to increase the efficiency of HDR and decrease that of NHEJ to improve the efficiency of precise knock-in genome editing. This review discusses the potential usage of such small molecules in the context of gene therapy and their drug-likeness, from a medicinal chemist's perspective.

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