Browse

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

Cited 18 time in Web of Science Cited 22 time in Scopus
Issue Date
2019-08
Citation
Molecular Therapy, Vol.27 No.8, pp.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.
ISSN
1525-0016
URI
https://hdl.handle.net/10371/165711
DOI
https://doi.org/10.1016/j.ymthe.2019.05.013
Files in This Item:
There are no files associated with this item.
Appears in Collections:
Seoul National University(서울대학교)Featured Researcher's Articles
  • mendeley

Items in S-Space are protected by copyright, with all rights reserved, unless otherwise indicated.

Browse