McLean, Zachariah L.
Gao, Dadi http://orcid.org/0000-0002-3638-8479
Correia, Kevin
Roy, Jennie C. L. http://orcid.org/0000-0003-3968-9637
Shibata, Shota http://orcid.org/0000-0002-9131-5408
Farnum, Iris N. http://orcid.org/0000-0003-2189-4579
Valdepenas-Mellor, Zoe
Kovalenko, Marina
Rapuru, Manasa
Morini, Elisabetta http://orcid.org/0000-0002-1658-5213
Ruliera, Jayla
Gillis, Tammy
Lucente, Diane
Kleinstiver, Benjamin P. http://orcid.org/0000-0002-5469-0655
Lee, Jong-Min http://orcid.org/0000-0001-5799-0787
MacDonald, Marcy E.
Wheeler, Vanessa C.
Mouro Pinto, Ricardo http://orcid.org/0000-0001-6744-2805
Gusella, James F. http://orcid.org/0000-0003-0681-9263
Funding for this research was provided by:
U.S. Department of Health & Human Services | NIH | National Institute of Neurological Disorders and Stroke (NS091161, NS105709, NS119471, NS049206, NS126420)
CHDI Foundation
Hereditary Disease Foundation
U.S. Department of Health & Human Services | NIH | National Cancer Institute (DP2-CA281401)
U.S. Department of Health & Human Services | NIH | National Institute of Neurological Disorders and Stroke
U.S. Department of Health & Human Services | NIH | National Institute of Neurological Disorders and Stroke
U.S. Department of Health & Human Services | NIH | National Institute of Neurological Disorders and Stroke
U.S. Department of Health & Human Services | NIH | National Institute of Neurological Disorders and Stroke
Huntington’s Disease Society of America
Article History
Received: 24 July 2023
Accepted: 30 March 2024
First Online: 12 April 2024
Competing interests
: J.F.G. and V.C.W. were founding scientific advisory board members with a financial interest in Triplet Therapeutics Inc. Their financial interests were reviewed and are managed by Massachusetts General Hospital (MGH) and Mass General Brigham (MGB) in accordance with their conflict of interest policies. J.F.G. consults for Transine Therapeutics, Inc. (dba Harness Therapeutics) and has previously provided paid consulting services to Wave Therapeutics USA Inc., Biogen Inc. and Pfizer Inc. V.C.W. is a scientific advisory board member of LoQus23 Therapeutics Ltd. and has provided paid consulting services to Acadia Pharmaceuticals Inc., Alnylam Inc., Biogen Inc., Passage Bio and Rgenta Therapeutics. R.M.P. and V.C.W. have received research support from Pfizer Inc. B.P.K. is a consultant for EcoR1 capital and Novartis Venture Fund, and is on the scientific advisory board of Acrigen Biosciences, Life Edit Therapeutics and Prime Medicine. B.P.K. has a financial interest in Prime Medicine, Inc., 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. J-M.L. consults for Life Edit Therapeutics and serves on the scientific advisory board of GenEdit Inc. E.M. is inventor on an International Patent Application Number PCT/US2021/012103, assigned to Massachusetts General Hospital and PTC Therapeutics entitled “RNA Splicing Modulation” related to use of BPN-15477 in modulating splicing. E.M. is a scientific advisory board member of ReviR Therapeutics, Inc and has received research support from PTC Therapeutics, Inc. The other authors declare no competing interests.