Kim, Jinkuk http://orcid.org/0000-0003-0564-3294
Woo, Sijae
de Gusmao, Claudio M.
Zhao, Boxun http://orcid.org/0000-0003-2337-5756
Chin, Diana H. http://orcid.org/0000-0002-1645-3552
DiDonato, Renata L.
Nguyen, Minh A.
Nakayama, Tojo http://orcid.org/0000-0003-2110-6878
Hu, Chunguang April
Soucy, Aubrie http://orcid.org/0000-0001-8633-0032
Kuniholm, Ashley
Thornton, Jennifer Karlin
Riccardi, Olivia
Friedman, Danielle A.
El Achkar, Christelle Moufawad
Dash, Zane http://orcid.org/0009-0002-7536-9622
Cornelissen, Laura
Donado, Carolina
Faour, Kamli N. W.
Bush, Lynn W.
Suslovitch, Victoria
Lentucci, Claudia
Park, Peter J. http://orcid.org/0000-0001-9378-960X
Lee, Eunjung Alice http://orcid.org/0000-0002-6574-9261
Patterson, Al
Philippakis, Anthony A.
Margus, Brad http://orcid.org/0009-0009-5447-6630
Berde, Charles B.
Yu, Timothy W. http://orcid.org/0000-0003-2988-7701
Article History
Received: 22 March 2022
Accepted: 25 May 2023
First Online: 12 July 2023
Competing interests
: J.K. and T.W.Y. are listed as inventors on pending patent claims (US 62/912,997) filed by Boston Children’s Hospital covering <i>ATM</i> splice-switching ASOs described in this study. T.W.Y. received research funding from ATCP and EveryOne Medicines, has served as a scientific consultant to Biomarin, GeneTx, Alnylam and Servier Pharmaceuticals and is a volunteer scientific advisor to several nonprofit rare disease foundations. B.M. is a co-founder, chairman and volunteer president of ATCP. J.K.T. is an employee of ATCP. A.A.P. is a Venture Partner at GV, a corporate venture group within Alphabet that invests in a number of rare disease drug development organizations, including EveryOne Medicines. The remaining authors declare no competing interests.