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Authors
Alves, Christiano R. R. https://orcid.org/0000-0002-2646-9689
Das, Sabyasachi
Krishnan, Vijai
Ha, Leillani L. https://orcid.org/0000-0003-4478-660X
Fox, Lauren R.
Stutzman, Hannah E.
Shamber, Claire E.
Kalailingam, Pazhanichamy
McCarthy, Siobhan
Lino Cardenas, Christian L. https://orcid.org/0000-0001-5491-4375
Fong, Claire E.
Imai, Takahiko
Mitra, Sunayana
Yun, Shuqi
Wood, Rachael K.
Benning, Friederike M. C.
Roh, Kangsan
Lawton, Joseph
Kim, Nahye
Silverstein, Rachel A.
Ferreira da Silva, Joana
de la Cruz, Demitri
Richa, Rashmi
Xie, Jun https://orcid.org/0000-0001-9565-1567
Gray-Edwards, Heather L. https://orcid.org/0000-0002-7856-3159
Malhotra, Rajeev https://orcid.org/0000-0003-0120-4630
Chung, David Y. https://orcid.org/0000-0002-7149-5851
Chao, Luke H. https://orcid.org/0000-0002-4849-4148
Tsai, Shengdar Q. https://orcid.org/0000-0001-9161-3993
Maguire, Casey A. https://orcid.org/0000-0001-8681-5179
Lindsay, Mark E. https://orcid.org/0000-0001-6724-7938
Kleinstiver, Benjamin P. https://orcid.org/0000-0002-5469-0655
Musolino, Patricia L. https://orcid.org/0000-0001-8724-5649
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Funding
Funding for this research was provided by:
U.S. Department of Health & Human Services | National Institutes of Health (P01HL142494, R01NS125353, DP2CA281401, K01NS134784, R01NS125353, R01NS125353)
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Article History
Received: 3 November 2024
Accepted: 6 August 2025
First Online: 11 September 2025
Competing interests
: C.L.L.C., R.M., C.A.M., D.Y.C., B.P.K., M.E.L. and P.L.M. are inventors on a patent application filed by MGB that describes the development of genome-editing technologies to treat MSMDS. C.R.R.A., R.A.S., J.F.d.S. and B.P.K. are inventors on additional patents or patent applications filed by MGB that describe genome engineering technologies. C.R.R.A. is a consultant for Biogen and Ilios Therapeutics. S.Q.T. is an inventor on a patent covering CHANGE-seq. S.Q.T. is a member of the scientific advisory board of Prime Medicine and Ensoma. R.M., D.Y.C., C.A.M., B.P.K., M.E.L. and P.L.M. received sponsored research support from Angea Biotherapeutics, a company developing gene therapies for vasculopathies. R.M. receives research funding from Amgen, serves as a consultant for Pharmacosmos, Myokardia/BMS, Renovacor, Epizon Pharma and Third Pole, and performs speaker bureaus through Vox Media, all of which are unrelated to the current work. C.A.M. has financial interests in Chameleon Biosciences, Skylark Bio and Sphere Gene Therapeutics, companies developing adeno-associated virus vector technologies for gene therapy applications; C.A.M. performs paid consulting work for all three companies. C.A.M.’s interests were reviewed and are managed by MGH and MGB in accordance with their conflict-of-interest policies. B.P.K. is a consultant for EcoR1 capital, Novartis Venture Fund, Foresite Labs and Jumble Therapeutics, and is on the scientific advisory boards of Acrigen Biosciences, Life Edit Therapeutics and Prime Medicine. B.P.K. has a financial interest in Prime Medicine, a company developing therapeutic CRISPR–Cas technologies for gene editing. B.P.K.’s interests were reviewed and are managed by MGH and MGB in accordance with their conflict-of-interest policies. The other authors declare no competing interests.
