Gao, Dadi http://orcid.org/0000-0002-3638-8479
Morini, Elisabetta http://orcid.org/0000-0002-1658-5213
Salani, Monica
Krauson, Aram J. http://orcid.org/0000-0001-7948-5730
Chekuri, Anil http://orcid.org/0000-0002-0652-2630
Sharma, Neeraj
Ragavendran, Ashok
Erdin, Serkan http://orcid.org/0000-0001-6587-2625
Logan, Emily M.
Li, Wencheng
Dakka, Amal
Narasimhan, Jana
Zhao, Xin
Naryshkin, Nikolai
Trotta, Christopher R.
Effenberger, Kerstin A.
Woll, Matthew G.
Gabbeta, Vijayalakshmi
Karp, Gary
Yu, Yong
Johnson, Graham
Paquette, William D. http://orcid.org/0000-0001-8427-7231
Cutting, Garry R. http://orcid.org/0000-0002-8250-8590
Talkowski, Michael E.
Slaugenhaupt, Susan A. http://orcid.org/0000-0001-7958-6494
Funding for this research was provided by:
Foundation for the National Institutes of Health (R21NS095437, R37NS095640)
Article History
Received: 28 February 2020
Accepted: 7 May 2021
First Online: 7 June 2021
Competing interests
: W.L., A.D., N.A.N., C.T., K.E., M.W., G.K., Y.Y., V.G., J.N., and X.Z. are employees of PTC Therapeutics, Inc., a biotechnology company. In connection with such employment, the authors receive salary, benefits and stock-based compensation, including stock options, restricted stock, other stock-related grants, and the right to purchase discounted stock through PTC’s employee stock purchase plan. Personal financial interests: S.A.S. is a paid consultant to PTC Therapeutics and is an inventor on several U.S. and foreign patents and patent applications assigned to the Massachusetts General Hospital, including U.S. Patents 8,729,025 and 9,265,766, both entitled “Methods for altering mRNA splicing and treating familial dysautonomia by administering benzyladenine,” filed on August 31, 2012 and May 19, 2014 and related to use of kinetin. S.A.S., G.J., and W.D.P. are inventors on U.S. Patent 10,675,475, assigned to Massachusetts General Hospital and entitled “Compounds for improving mRNA splicing” filed on July 14, 2017 and related to use of BPN-15477. D.G., E.M., W.L., K.A.E., C.R.T., Y.Y., V.G., A.D., N.A.N., M.E.T., and S.A.S. are inventors 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. All other authors declare no competing interests.