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

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[EZ-Cytox] CRISPR-Pass: Gene Rescue of Nonsense Mutations Using Adenine Base Editors

김상진
2020-01-08 15:34 6,569 1

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년도
2019
제품명
EZ-Cytox
학술지명
Molecular Therapy

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.

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김상진
2020-01-08 15:34
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