Journal
MOLECULAR THERAPY-NUCLEIC ACIDS
Volume 21, Issue -, Pages 1062-1073Publisher
CELL PRESS
DOI: 10.1016/j.omtn.2020.07.037
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Funding
- National Key Research and Development Program of China Stem Cell and Translational Research [2019YFA0110700, 2017YFA0105101]
- Program for Changjiang Scholars and Innovative Research Team in University [IRT_16R32]
- Strategic Priority Research Program of the Chinese Academy of Sciences [XDA16030501, XDA16030503]
- Key Research & Development Program of Guangzhou Regenerative Medicine and Health Guangdong Laboratory [2018GZR110104004]
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CRISPR-Cas9-mediated gene knockout and base-editing associated induction of STOP codons (iSTOP) have been widely used to exterminate the function of a coding gene, while they have been reported to exhibit side effects. In this study, we propose a novel and practical alternative method referred to as CRISPR Start-Loss (CRISPR-SL), which eliminates gene expression by utilizing both adenine base editors (ABEs) and cytidine base editors (CBEs) to disrupt the initiation codon (ATG). CRISPR-SL has been verified to be a feasible strategy on the cellular and embryonic levels (mean editing efficiencies up to 30.67% and 73.50%, respectively) and in two rabbit models mimicking Otc deficiency (Otc gene) and long hair economic traits (Fgf5 gene).
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