Clinical and Experimental Pediatrics

대한소아청소년과학회 (The Korean Pediatric Society)
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Application of CRISPR-Cas9 gene editing for congenital heart disease

  • Seok, Heeyoung (Department of Life Sciences, Korea University) ;
  • Deng, Rui (Department of Cardiology, Boston Children's Hospital) ;
  • Cowan, Douglas B. (Department of Cardiology, Boston Children's Hospital) ;
  • Wang, Da-Zhi (Department of Cardiology, Boston Children's Hospital)
  • 투고 : 2020.12.27
  • 심사 : 2021.02.15
  • 발행 : 2021.06.15
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초록

Clustered regularly interspaced short palindromic repeats and CRISPR-associated protein 9 (CRISPR-Cas9) is an ancient prokaryotic defense system that precisely cuts foreign genomic DNA under the control of a small number of guide RNAs. The CRISPR-Cas9 system facilitates efficient double-stranded DNA cleavage that has been recently adopted for genome editing to create or correct inherited genetic mutations causing disease. Congenital heart disease (CHD) is generally caused by genetic mutations such as base substitutions, deletions, and insertions, which result in diverse developmental defects and remains a leading cause of birth defects. Pediatric CHD patients exhibit a spectrum of cardiac abnormalities such as septal defects, valvular defects, and abnormal chamber development. CHD onset occurs during the prenatal period and often results in early lethality during childhood. Because CRISPR-Cas9-based genome editing technology has gained considerable attention for its potential to prevent and treat diseases, we will review the CRISPR-Cas9 system as a genome editing tool and focus on its therapeutic application for CHD.

키워드

과제정보

This work is supported by NRF-2020R1A2C1013377, NRF-2017R1D1A1B03030852 for H Seok and HL149401 from NIH to DZ Wang. We thank members of the Wang Laboratory for their careful reading and comments.

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피인용 문헌

  1. Hypertrophic Cardiomyopathy in Infants from the Perspective of Cardiomyocyte Maturation vol.51, pp.9, 2021, https://doi.org/10.4070/kcj.2021.0153