Authorship: In addition to Musolino, Kleinstiver and Lindsay, Mass General Brigham authors include Christiano R. R. Alves, Sabyasachi Das, Vijai Krishnan, Leillani L. Ha, Lauren R. Fox, Hannah E. Stutzman, Claire E. Shamber, Pazhanichamy Kalailingam, Siobhan McCarthy, Christian L. Lino Cardenas, Claire E. Fong, Takahiko Imai, Sunayana Mitra, Shuqi Yun, Rachael K. Wood, Kangsan Roh, Joseph Lawton, Nahye Kim, Rachel A. Silverstein, Joana Ferreira da Silva, Demitri de la Cruz, Rashmi Richa, Jun Xie, Heather L. Gray-Edwards, Rajeev Malhotra, David Y. Chung, Luke H. Chao, and Casey A. Maguire. Additional authors include Friederike M. C. Benning, Shengdar Q. Tsai.
Disclosures: Lino Cardenas, Malhotra, Maguire, Chung, Kleinstiver, Lindsay, and Musolino are inventors on a patent application filed by MGB that describes the development of genome-editing technologies to treat MSMDS. Alves, Silverstein, Ferreira da Silva, and Kleinstiver are inventors on additional patents or patent applications filed by MGB that describe genome engineering technologies. Tsai is an inventor on a patent covering CHANGE-seq and is a member of the scientific advisory board of Prime Medicine and Ensoma. Malhotra, Chung, Maguire, Kleinstiver, Lindsay, and Musolino received sponsored research support from Angea Biotherapeutics, a company developing gene therapies for vasculopathies. Musolino receives research funding from Minoryx, serves as a consultant for Vertex, Astellas, Biogen, Ionis, Altas Venture, Inozyme, all of which are unrelated to the current work. Malhotra 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. Maguire has financial interests in Chameleon Biosciences, Skylark Bio, and Sphere Gene Therapeutics, companies developing adeno-associated virus vector technologies for gene therapy applications, and performs paid consulting work for all three companies. Maguire’s interests were reviewed and are managed by MGH and MGB in accordance with their conflict-of-interest policies. Kleinstiver 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. Kleinstiver has a financial interest in Prime Medicine, a company developing therapeutic CRISPR–Cas technologies for gene editing, and Kleinstiver’s interests were reviewed and are managed by MGH and MGB in accordance with their conflict-of-interest policies.
Funding: This study was funded in part by Charles A. King Trust Postdoctoral Research Fellowship, Bank of America, N.A., Co-Trustees, James L. and Elisabeth C. Gamble Endowed Fund for Neuroscience Research/Mass General Neuroscience Transformative Scholar Award, MGH Physician/Scientist Development Award, Natural Sciences and Engineering Research Council of Canada Postgraduate Scholarship–Doctoral (PGS D – 567791), EMBO Long Term Fellowship (ALTF 750-2022), Swiss National Science Foundation (P180777), St. Jude Children’s Research Hospital and ALSAC, National Institutes of Allergy and Infectious Diseases (U01AI176470, U01AI176471), MGH Howard M. Goodman Fellowship, Kayden–Lambert MGH Research Scholar Award 2023-2028, Angea Biotherapeutics (sponsored research agreement), National Institutes of Health (K01NS134784, R01HL162928, K08NS112601, R35GM142553, DC017117, DP2CA281401, P01HL142494, R01NS125353).
Paper cited: Alves CRR et al. “Treatment of a severe vascular disease using a bespoke CRISPR–Cas9 base editor in mice” Nature Biomedical Engineering DOI: 10.1038/s41551-025-01499-1