CRISPR-Pass: Gene Rescue of Nonsense Mutations Using Adenine Base Editors

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Title
CRISPR-Pass: Gene Rescue of Nonsense Mutations Using Adenine Base Editors
Author(s)
Choongil Lee; Dong Hyun Jo; Gue-Ho Hwang; Jihyeon Yu; Jin Hyoung Kim; Se-eun Park; Jin Soo Kim; Jeong Hun Kim; Sangsu Bae
Publication Date
2019-08
Journal
MOLECULAR THERAPY, v.27, no.8, pp.1364 - 1371
Publisher
CELL PRESS
Abstract
© 2019 The Author(s)A nonsense mutation is a substitutive mutation in a DNA sequence that causes a premature termination during translation and produces stalled proteins, resulting in dysfunction of a gene. Although it usually induces severe genetic disorders, there are no definite methods for inducing read through of premature termination codons (PTCs). Here, we present a targeted tool for bypassing PTCs, named CRISPR-pass, that uses CRISPR-mediated adenine base editors. CRISPR-pass, which should be applicable to 95.5% of clinically significant nonsense mutations in the ClinVar database, rescues protein synthesis in patient-derived fibroblasts, suggesting potential clinical utility. Lee et al. showed that CRISPR-pass, based on adenine base editors, would be a targeted tool for bypassing premature termination codons. This system could be applicable to ∼95% of clinically significant nonsense mutations, related to genetic diseases, in the ClinVar database
URI
https://pr.ibs.re.kr/handle/8788114/6097
ISSN
1525-0016
Appears in Collections:
Center for Genome Engineering(유전체 교정 연구단) > Journal Papers (저널논문)
Files in This Item:
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