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나노의학 연구단
나노의학 연구단
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In vivo gene correction with targeted sequence substitution through microhomology-mediated end joining

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Title
In vivo gene correction with targeted sequence substitution through microhomology-mediated end joining
Author(s)
Jeong Hong Shin; Soobin Jung; Suresh Ramakrishna; Hyongbum Henry Kim; Junwon Lee
Publication Date
2018-07
Journal
BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS, v.502, no.1, pp.116 - 122
Publisher
ACADEMIC PRESS INC ELSEVIER SCIENCE
Abstract
Genome editing technology using programmable nucleases has rapidly evolved in recent years. The primary mechanism to achieve precise integration of a transgene is mainly based on homology-directed repair (HDR). However, an HDR-based genome-editing approach is less efficient than non-homologous end-joining (NHEJ). Recently, a microhomology-mediated end-joining (MMEJ)-based transgene integration approach was developed, showing feasibility both in vitro and in vivo. We expanded this method to achieve targeted sequence substitution (TSS) of mutated sequences with normal sequences using double-guide RNAs (gRNAs), and a donor template flanking the microhomologies and target sequence of the gRNAs in vitro and in vivo. Our method could realize more efficient sequence substitution than the HDR-based method in vitro using a reporter cell line, and led to the survival of a hereditary tyrosinemia mouse model in vivo. The proposed MMEJ-based TSS approach could provide a novel therapeutic strategy, in addition to HDR, to achieve gene correction from a mutated sequence to a normal sequence. © 2018 Elsevier Inc
URI
https://pr.ibs.re.kr/handle/8788114/5441
ISSN
0006-291X
Appears in Collections:
Center for Nanomedicine (나노의학 연구단) > Journal Papers
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