Article
Co-opting regulation bypass repair as a gene-correction strategy for monogenic diseases.
Molecular therapy : the journal of the American Society of Gene Therapy - 3 Nov 2021
Hu Jingjie, Bourne Rebecca A, McGrath Barbara C, Lin Alice, Pei Zifei, Cavener Douglas R
Abstract excerpt
With the development of CRISPR-Cas9-mediated gene-editing technologies, correction of disease-causing mutations has become possible. However, current gene-correction strategies preclude mutation repair in post-mitotic cells of human tissues, and a unique repair strategy must be designed and tested for each and every mutation that may occur in a gene. We have developed a novel gene-correction strategy, co-opting...
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