Original paper
Allele specific repair of splicing mutations in cystic fibrosis through AsCas12a genome editing
Abstract
Cystic fibrosis (CF) is an autosomal recessive disease caused by mutations in the CFTR gene. The 3272-26A>G and 3849+10kbC>T CFTR mutations alter the correct splicing of the CFTR gene, generating new acceptor and donor splice sites respectively. Here we develop a genome editing approach to permanently correct these genetic defects, using a single crRNA and the Acidaminococcus sp. BV3L6, AsCas12a. This genetic repair strategy is highly precise,...
Paper Details
Title
Allele specific repair of splicing mutations in cystic fibrosis through AsCas12a genome editing
Published Date
Aug 7, 2019
Journal
Volume
10
Issue
1
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Notes
History