News
Photoreceptor transplants restore vision in mice
Key achievements
The world's first eye gene therapy trial - JWB Bainbridge et al. N Engl J Med. 2008
Cell therapy repairs the retina - Pearson RA et al Nature 2012
The first proof-of-concept in eye gene therapy - RR Ali et al. Nat. Genet. 2000
Our latest blog posts
It’s OK to ask about clinical research – an NIHR campaign
‘Defining future eye research’ – a chance for you to help tackle slight loss
Clinical trials
Effect
of gene therapy on visual function in adults with Leber Congenital Amaurosis. JWB
Bainbridge, AJ Smith, SE Barker, S Robbie, R Henderson, KS Balaggan, A
Viswanathan, GE Holder, A Stockman, N Tyler, S Petersen-Jones, SS Bhattacharya,
AJ Thrasher, FW Fitzke, BJ Carter, GS Rubin, AT Moore and RR Ali N
Engl J Med. 2008; 358 (21): 2231-9
Gene therapy of X-linked
severe combined immunodeficiency by use of a pseudotyped gammaretroviral
vector. HB Gaspar, KL Parsley, S Howe, D King , KC Gilmour, J
Sinclair, G Brouns, M Schmidt, C Von Kalle, T Barington, M Jakobsen, HO
Christensen, A Al Ghonaium, HN White, JL Smith, RJ Levinsky, RR Ali, C Kinnon
and AJ Thrasher Lancet 2004; 364 (9452):
2181-2187
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