Open AccessBase editing rescue of spinal muscular atrophy in cells and in mice
Author(s) -
Mandana Arbab,
Żaneta Matuszek,
Kaitlyn M. Kray,
Ailing Du,
Gregory A. Newby,
Anton J. Blatnik,
Aditya Raguram,
Michelle F. Richter,
Kevin T. Zhao,
Jonathan M. Levy,
Max W. Shen,
W. David Arnold,
Dan Wang,
Jun Xie,
Guangping Gao,
Arthur H.M. Burghes,
David R. Liu
Publication year - 2023
Publication title -
science
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 12.556
H-Index - 1186
eISSN - 1095-9203
pISSN - 0036-8075
DOI - 10.1126/science.adg6518
Subject(s) - smn1 , spinal muscular atrophy , sma* , genome editing , biology , motor neuron , mutation , phenotype , genetics , cancer research , gene , neuroscience , genome , spinal cord , computer science , algorithm
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