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

Title
CRISPR-Pass: Gene Rescue of Nonsense Mutations Using Adenine Base Editors
Author
유지현
Keywords
GENOMIC DNA; CELLS
Issue Date
2019-08
Publisher
CELL PRESS
Citation
MOLECULAR THERAPY, v. 27, NO 8, Page. 1364-1371
Abstract
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.
URI
https://www.sciencedirect.com/science/article/pii/S1525001619302278?via%3Dihubhttps://repository.hanyang.ac.kr/handle/20.500.11754/111251
ISSN
1525-0016; 1525-0024
DOI
10.1016/j.ymthe.2019.05.013
Appears in Collections:
RESEARCH INSTITUTE[S](부설연구소) > ETC
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