Correction of a splicing defect in a mouse model of congenital muscular dystrophy type 1A using a homology-directed-repair-independent mechanism

Dwi U Kemaladewi,Eleonora Maino,Elzbieta Hyatt,Huayun Hou,Maylynn Ding, Kara M Place,Xinyi Zhu,Prabhpreet Bassi,Zahra Baghestani,Amit G Deshwar,Daniele Merico,Hui Y Xiong,Brendan J Frey,Michael D Wilson,Evgueni A Ivakine,Ronald D Cohn

NATURE MEDICINE（2017）

引用 67|浏览61

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摘要

n HDR-independent therapeutic genome-editing approach corrected the splice-site mutation in Lama2 in a mouse model of congenital muscular dystrophy type 1A, and may be applied more broadly to correct splice-site mutations associated with other diseases.

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关键词

Neuromuscular disease,RNA splicing,Biomedicine,general,Cancer Research,Metabolic Diseases,Infectious Diseases,Molecular Medicine,Neurosciences

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