Article
Multiple isogenic GNE-myopathy modeling with mutation specific phenotypes from human pluripotent stem cells by base editors.
Biomaterials - 1 Mar 2022
Park Ju-Chan, Kim Jumee, Jang Hyun-Ki, Lee Seung-Yeon, Kim Keun-Tae, Kwon Eun-Ji, Park Seokwoo, Lee Hyun Sik, Choi Hyewon, Park Seung-Yeol, Choi Hee-Jung, Park Soon-Jung, Moon Sung-Hwan, Bae Sangsu, Cha Hyuk-Jin
Abstract excerpt
Despite the great potential of disease modeling using human pluripotent stem cells (hPSCs) derived from patients with mutations, lack of an appropriate isogenic control hinders a precise phenotypic comparison due to the bias arising from the dissimilar genetic backgrounds between the control and diseased hPSCs. Herein, we took advantage of currently available base editors (BEs) to epitomize the isogenic disease...
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