@article{9e427824c52744dd8300cace7c6e4c2e,
title = "Treatment of a Mouse Model of ALS by In Vivo Base Editing",
abstract = "Lim et al. establish a trans-splicing system to deliver CRISPR base editors in vivo that enables treatment of a mouse model of ALS. They show that base editing can increase survival and slow the progression of disease.",
keywords = "AAV, ALS, base editor, CRISPR-Cas9, gene editing, gene therapy, neurodegeneration, SOD1, split intein",
author = "Lim, {Colin K.W.} and Michael Gapinske and Brooks, {Alexandra K.} and Woods, {Wendy S.} and Powell, {Jackson E.} and {Zeballos C.}, {M. Alejandra} and Jackson Winter and Pablo Perez-Pinera and Thomas Gaj",
note = "Funding Information: We thank P. Jensen and S. Sirk for helpful discussion. C.K.W.L. was supported by a Roy J. Carver Fellowship in Engineering. M.G. was supported by a National Science Foundation Graduate Research Fellowship; M.A.Z.C. was supported by a University of Illinois Aspire Fellowship. This work was supported by the Muscular Dystrophy Association, United States (MDA602798 to T.G. and P.P.-P.), the Judith and Jean Pape Adams Foundation, United States (to T.G.), the American Heart Association, United States (17SDG33650087 to P.P.-P.), and by the National Institutes of Health, United States (R01GM127497 to P.P.-P.). Publisher Copyright: {\textcopyright} 2020 The American Society of Gene and Cell Therapy",
year = "2020",
month = apr,
day = "8",
doi = "10.1016/j.ymthe.2020.01.005",
language = "English (US)",
volume = "28",
pages = "1177--1189",
journal = "Molecular Therapy",
issn = "1525-0016",
publisher = "Cell Press",
number = "4",
}