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EyeTherapy Blog News

UCL Gene Therapy Trial shows Benefit for up to Three Years After Treatment

Tue, 05 May 2015 14:44:39 +0000

We are delighted to be able to announce that yesterday, Monday 4th May, the long-term results of our RPE65 gene therapy trial for Leber Congenital Amaurosis Type 2 (LCA2) were published in the prestigious New England Journal of Medicine. Begun in 2007, this was the world’s first-in-human trial of gene therapy to treat an inherited […]

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UCL researchers solve a major riddle of retinal degeneration research!

Mon, 26 Jan 2015 10:11:36 +0000

Today a paper published in Nature Communications from the Gene and Cell Therapy Group at the UCL Institute of Ophthalmology has shed light on why, until now, it has not been possible to effectively restore vision in rd1 mice – the world’s major model for retinitis pigmentosa (RP). The rd1 mouse is a model of […]

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Professor Ali honoured for his contribution to research into retinal disease

Mon, 08 Sep 2014 12:27:36 +0000

              Professor Robin Ali, PhD, Professor of Human Molecular Genetics and Head of the Department of Genetics, UCL Institute of Opthalmology has been awarded the Pioneer Award for his work in proof-of-concept studies that have demonstrated the feasibility of using gene therapy and cell transplantation to treat dysfunction and degeneration of the cells […]

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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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