Mayuranathan, Thiyagaraj http://orcid.org/0000-0002-9505-0363
Newby, Gregory A. http://orcid.org/0000-0001-7869-2615
Feng, Ruopeng
Yao, Yu
Mayberry, Kalin D.
Lazzarotto, Cicera R. http://orcid.org/0000-0003-1093-0175
Li, Yichao http://orcid.org/0000-0001-5791-096X
Levine, Rachel M. http://orcid.org/0000-0002-3684-2950
Nimmagadda, Nikitha http://orcid.org/0000-0003-1648-6335
Dempsey, Erin http://orcid.org/0009-0000-2512-0939
Kang, Guolian http://orcid.org/0000-0002-6102-0115
Porter, Shaina N.
Doerfler, Phillip A. http://orcid.org/0000-0001-7566-9073
Zhang, Jingjing
Jang, Yoonjeong
Chen, Jingjing http://orcid.org/0000-0003-4179-0371
Bell, Henry W. http://orcid.org/0000-0003-4677-8208
Crossley, Merlin http://orcid.org/0000-0003-2057-3642
Bhoopalan, Senthil Velan http://orcid.org/0000-0002-1402-6549
Sharma, Akshay http://orcid.org/0000-0003-3281-2081
Tisdale, John F.
Pruett-Miller, Shondra M. http://orcid.org/0000-0002-3793-585X
Cheng, Yong http://orcid.org/0000-0002-9314-0971
Tsai, Shengdar Q. http://orcid.org/0000-0001-9161-3993
Liu, David R. http://orcid.org/0000-0002-9943-7557
Weiss, Mitchell J. http://orcid.org/0000-0003-2460-3036
Yen, Jonathan S. http://orcid.org/0000-0002-9432-9450
Funding for this research was provided by:
U.S. Department of Health & Human Services | NIH | National Heart, Lung, and Blood Institute (HL156647, HL136135, HL053749, HL163805, HL053749, HL156647, HL136135, HL156647, HL136135, HL053749)
St. Jude Children’s Research Hospital
American Lebanese Syrian Associated Charities
Assisi Foundation of Memphis
Bill and Melinda Gates Foundation
Helen Hay Whitney Foundation
Cooley’s Anemia Foundation
U.S. Department of Health & Human Services | NIH | National Institute of Diabetes and Digestive and Kidney Diseases (K01 DK132453)
American Society of Hematology
Doris Duke Charitable Foundation
U.S. Department of Health & Human Services | NIH | National Institute of General Medical Sciences (GM133614, GM118062)
U.S. Department of Health & Human Services | NIH | National Institute of Allergy and Infectious Diseases (AI157189, AI142756)
U.S. Department of Health & Human Services | NIH | National Human Genome Research Institute (HG009490)
U.S. Department of Health & Human Services | NIH | National Institute of Biomedical Imaging and Bioengineering (EB022376)
Howard Hughes Medical Institute
Article History
Received: 11 July 2022
Accepted: 23 May 2023
First Online: 3 July 2023
Competing interests
: The authors have filed patent applications on genome-editing agents. D.R.L. is a consultant and equity owner of Beam Therapeutics, Prime Medicine, Pairwise Plants, Chroma Medicine and Nvelop Therapeutics, companies that use or deliver genome editing or genome engineering agents. M.J.W. is a consultant for GSK, Cellarity, Novartis and Dyne Therapeutics. A.S. is a consultant for Spotlight Therapeutics, Medexus., and Vertex Pharmaceuticals. A.S. has also received research funding from CRISPR Therapeutics and honoraria from Vindico Medical Education. A.S. is the St. Jude Children’s Research Hospital site principal investigator of clinical trials for genome editing of sickle cell disease sponsored by Vertex Pharmaceuticals/CRISPR Therapeutics (ExternalRef removed), Novartis Pharmaceuticals (ExternalRef removed) and Beam Therapeutics (ExternalRef removed). The industry sponsors provide funding for the clinical trial, which includes salary support. A.S. has no direct financial interest in these therapies. J.S.Y. is an equity owner of Beam Therapeutics. S.Q.T. is a co-inventor on patents covering the CIRCLE-seq and CHANGE-seq methods. S.Q.T. is a member of the scientific advisory boards of Kromatid and Twelve Bio. The remaining authors declare no competing interests.