Liu, Pengpeng http://orcid.org/0000-0002-4891-8780
Ponnienselvan, Karthikeyan http://orcid.org/0000-0002-8016-9433
Nyalile, Thomas
Oikemus, Sarah
Joynt, Anya T.
Iyer, Sukanya
Kelly, Karen
Guo, Dongsheng
Kyawe, Pyae P.
Vanderleeden, Emma http://orcid.org/0009-0005-5339-1863
Redick, Sambra D.
Huang, Lei http://orcid.org/0000-0002-0568-0645
Chen, Zexiang http://orcid.org/0000-0002-1584-2417
Lee, Jeong Min
Schiffer, Celia A. http://orcid.org/0000-0003-2270-6613
Harlan, David M.
Wang, Jennifer P. http://orcid.org/0000-0002-2556-9100
Emerson, Charles P. Jr
Lawson, Nathan D.
Watts, Jonathan K. http://orcid.org/0000-0001-5706-1734
Sontheimer, Erik J. http://orcid.org/0000-0002-0881-0310
Luban, Jeremy http://orcid.org/0000-0001-5650-4054
Wolfe, Scot A. http://orcid.org/0000-0002-7042-201X
Funding for this research was provided by:
U.S. Department of Health & Human Services | NIH | National Heart, Lung, and Blood Institute (R01HL150669, R35HL140017)
U.S. Department of Health & Human Services | NIH | National Center for Advancing Translational Sciences (UH3TR002668, UH3TR002668)
U.S. Department of Health & Human Services | NIH | National Institute of Allergy and Infectious Diseases (R37AI147868, R01AI150478, R37AI147868, U54AI170856)
Friedreich’s Ataxia Research Alliance Rett syndrome Research Trust
U.S. Department of Health & Human Services | NIH | Eunice Kennedy Shriver National Institute of Child Health and Human Development (U54HD0060848)
U.S. Department of Health & Human Services | National Institutes of Health (R21OD030004)
Fondation Leducq
U.S. Department of Health & Human Services | NIH | National Institute of General Medical Sciences (R01GM150273)
JDRF (COE-2020-967-M-N, COE-2020-967-M-N)
Article History
Received: 9 October 2023
Accepted: 27 August 2024
First Online: 25 September 2024
Competing interests
: E.J.S. is a cofounder and Scientific Advisory Board member of Intellia Therapeutics and a Scientific Advisory Board member at Tessera Therapeutics. S.A.W. is a consultant for Editas Medicine. The University of Massachusetts Chan Medical School has filed patent applications related to this work. All other authors have no competing interests.