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CRISPR-SKIP: programmable gene splicing with single base editors

delete2018-08-15
delete148
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OA
AI
M
Michael Gapinske
A
Alan Luu
J
Jackson Winter
W
Wendy S. Woods
K
Kurt A. Kostan
N
Nikhil Shiva
J
Jun S. Song *
P
Pablo Pérez‐Piñera *
DOI:10.1186/s13059-018-1482-5delete
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摘要

摘要

En 中文
CRISPR gene editing has revolutionized biomedicine and biotechnology by providing a simple means to engineer genes through targeted double-strand breaks in the genomic DNA of living cells. However, given the stochasticity of cellular DNA repair mechanisms and the potential for off-target mutations, technologies capable of introducing targeted changes with increased precision, such as single-base editors, are preferred. We present a versatile method termed CRISPR-SKIP that utilizes cytidine deaminase single-base editors to program exon skipping by mutating target DNA bases within splice acceptor sites. Given its simplicity and precision, CRISPR-SKIP will be broadly applicable in gene therapy and synthetic biology.
Keyword:
Gene editing
Base editing
Exon skipping
Alternative splicing
Synthetic biology
CRISPR-Cas9
RELA
PIK3CA
BRCA2
Gene isoform
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期刊

G
Genome Biology
IF:
9.4
论文数:
6.4K
被引数:
7.3W

机构

U
University of Illinois Urbana-Champaign
学者数:
2.4W
论文数: 2.0W
被引数: 35
University of Illinois System 封面图
University of Illinois System
学者数:
6.9W
论文数: 6.2W
被引数: 644
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