Publications
bioRxivJan 2021 DOI:
10.1101/2021.01.08.425835

Prime editing enables precise genome editing in mouse liver and retina

Jang, Hyewon; Shin, Jeong Hong; Jo, Dong Hyun; Seo, Jung Hwa; Yu, Goosang; Gopalappa, Ramu; Cho, Sung-Rae; Kim, Jeong Hun; Kim, Hyongbum Henry
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Oligo Pools
Abstract
Prime editing can induce any small-sized genetic change without donor DNA or double strand breaks. However, it has not been investigated whether prime editing is possible in postnatal animals. Here we delivered prime editors 2 and 3 into a mouse model of hereditary tyrosinemia, a genetic liver disease, using hydrodynamic injection, which corrected the disease-causing mutation and rescued the phenotype. We also achieved prime editing in the retina and retina pigment epithelium in wild-type mice by delivering prime editor 3 using trans-splicing adeno-associated virus. Deep sequencing showed that unintended edits at or near the target site or off-target effects were not detectable except for low levels (0% to 1.2%) of indels when PE3, but not PE2, was used. Our study suggests that precise, prime editor-mediated genome editing is possible in somatic cells of adult animals.
Product Used
Oligo Pools

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