Cehajic-Kapetanovic, Jasmina http://orcid.org/0000-0002-9956-6412
Xue, Kanmin
Martinez-Fernandez de la Camara, Cristina
Nanda, Anika
Davies, Alexandra
Wood, Laura J.
Salvetti, Anna Paola http://orcid.org/0000-0002-5513-2241
Fischer, M. Dominik
Aylward, James W.
Barnard, Alun R. http://orcid.org/0000-0002-0662-1073
Jolly, Jasleen K. http://orcid.org/0000-0001-9878-4621
Luo, Edmond
Lujan, Brandon J.
Ong, Tuyen
Girach, Aniz
Black, Graeme C. M.
Gregori, Ninel Z.
Davis, Janet L. http://orcid.org/0000-0001-6395-5881
Rosa, Potyra R.
Lotery, Andrew J. http://orcid.org/0000-0001-5541-4305
Lam, Byron L.
Stanga, Paulo E.
MacLaren, Robert E.
Funding for this research was provided by:
DH | National Institute for Health Research
Trial was sponsored by Biogen Inc.
Article History
Received: 20 May 2019
Accepted: 10 January 2020
First Online: 24 February 2020
Competing interests
: R.E.M. is the scientific cofounder of Nightstar Therapeutics Inc. (now owned by Biogen Inc.). R.E.M. and G.C.M.B. are scientific advisors to the UK’s National Institute for Health and Care Excellence in relation to retinal gene therapy. M.D.F., B.L.L., B.J.L. and R.E.M. are consultants or on the advisory board for Biogen Inc. R.E.M. and M.D.F. are the named inventors on the patent relating to codon-optimized <i>RPGR</i> gene therapy, owned by the University of Oxford (patent no. US20180273594A1, originally filed 10 September 2015). M.D.F., G.C.M.B. and R.E.M. are on the scientific advisory board to Novartis. E.L., T.O. and A.G. were employees of the sponsor of the trial, Nightstar Therapeutics (now Biogen Inc.). The views expressed are those of the authors and not necessarily those of the National Health Service, the NIHR or the UK Department of Health.