Eye Therapy News

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 […]


Achromatopsia might not be as progressive as previously thought

Mon, 08 Sep 2014 11:17:16 +0000

A recent publication from the UCL Institute of Ophthalmology, Moorfields Eye Hospital, and the Medical College of Wisconsin indicates that for the majority of people with achromatopsia, the condition may not be as progressive as previously suggested. Data from this study by Aboshiha et al. demonstrated that for the majority of people with achromatopsia (a […]


2 Lazy 2 Run? We’re biking it for blood cancer!

Fri, 29 Aug 2014 09:30:05 +0000

  On Sunday 31 August a group of not so elite athletes from the Gene and Cell Therapy group will be taking part in the London Bikeathon 2014 to raise funds for Leukaemia & Lymphoma Research. The 2 Lazy 2 Run CC will be cycling 52 miles – that’s more than a marathon, no mean feet […]


The Art of Eyes

Thu, 07 Aug 2014 14:23:19 +0000

The eye is an object of great beauty as shown by the Ophthalmologist in their July/August 2014 issue. This month’s issue features a photo essay called The Art of the Eyes and includes examples of the work from a number research labs capturing the complex and beautiful detail of the eye and its cells. The essay includes images […]


In memoriam

Tue, 05 Aug 2014 16:02:34 +0000

Dr Yoshiki Sasai (1962 – 2014) It is with great sadness today that we remember and pay tribute to our collaborator Dr Yoshiki Sasai. Yoshiki was a world leading stem cell researcher and Deputy Director of the Riken Center for Developmental Biology in Kobe, Japan. Through his hard work and dedication over many years, Yoshiki […]


Gene and cell therapies for inherited sight loss

Gene therapy in models of disease - proof-of-concept

Long-term and age-dependent restoration of visual function in a mouse model of CNGB3-associated achromatopsia following gene therapy.
LS Carvalho, J Xu, RA Pearson, AJ Smith, JW Bainbridge, LM Morries, SJ Fliesler, XQ Ding and RR Ali. Hum Mol Genet. 2011; 20 (16): 3161-75

Long-term preservation of cones and improvement in visual function following gene therapy in a mouse model of leber congenital amaurosis caused by guanylate cyclase-1 deficiency.
Mihelec M, Pearson RA, Robbie SJ, Buch PK, Azam SA, Bainbridge JW, Smith AJ, Ali RR. Hum Gene Ther. 2011 Oct;22 (10):1179-90

Gene Therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/8 vectors.
MH Tan, AJ Smith, B Pawlyk, X Xu, X Liu, JW Bainbridge, M Basche, J McIntosh, HV Tran, A Nathanwi, T Li and RR Ali Hum Mol Genet. 2009; 18 (12): 2099-114

AAV-mediated knockdown of Peripherin-2 in vivo using miRNA-based hairpins.
A Georgiadis, M Tschernutter, JW Bainbridge, SJ Robbie, J McIntosh, AC Nathwani, AJ Smith and RR Ali Gene Ther. 2010;17 (4): 486-93

In contrast to AAV-mediated CNTF expression, AAV-mediated expression of GDNF enhances gene replacement therapy in rodent models of retinal degeneration.
PK Buch, RE MacLaren, Y Durán, KS Balaggan, A MacNeil, FC Schlichtenbrede, AJ Smith and RR Ali Mol. Ther. 2006; 14 (5): 700-709

Long term preservation of retinal function in the RCS rat model of retinitis pigmentosa following lentivirus-mediated gene therapy.
M Tschernutter, FC Schlichtenbrede, S Howe, KS Balaggan, PM Munro, JW Bainbridge, AJ Thrasher, AJ Smith and RR Ali Gene Ther. 2005; 12 (8): 694-701

Restoration of photoreceptor ultrastructure and function in retinal degeneration slow mice by gene therapy.
RR Ali, G Sarra, C Stephens, M de Alwis, JWB Bainbridge, PM Munro, S Fauser, MB Reichel, C Kinnon, DM Hunt, SS Bhattacharya and AJ Thrasher Nat. Genet. 2000; 25 (3): 306-310

Cell therapy

Restoration of vision after transplantation of photoreceptors
Pearson RA, Barber AC, Rizzi M, Hippert C, Xue T, West EL, Duran Y, Smith AJ, Chuang JZ, Azam SA, Luhmann UF, Benucci A, Sung CH, Bainbridge JW, Carandini M, Yau KW, Sowden JC, Ali RR. Nature 2012; 485 (7396):99-103.

Retinal repair by transplantation of photoreceptor precursors.
RE MacLaren, RA Pearson, A MacNeil, RH Douglas, TE Salt, M Akimoto, A Swaroop, JC Sowden and RR Ali Nature 2006; 444 (7116): 203-207

Cone and rod photoreceptor transplantation in models of the childhood retinopathy Leber congenital amaurosis using flow-sorted Crx-positive donor cells.
J Lakowski, M Baron, J Bainbridge, AC Barber, RA Pearson, RR Ali and JC Sowden. Hum Mol Genet. 2010; 19 (23): 4545-59

Gene therapy - improving gene delivery to the eye

Assessment of ocular transduction using single stranded and self-complementary recombinant adeno-associated virus serotype 2-8.
M Natkunaraja, P Trittibach, J McIntosh, Y Duran, SE Barker, AJ Smith, AC Nathwani and RR Ali Gene Ther. 2008; 15 (6): 463-7

Effective gene therapy with non-integrating lentiviral vectors.
RJ Yáñez-Muñoz, KS Balaggan, A MacNeil, S Howe, M Schmidt, AJ Smith, P Buch, RE MacLaren, PN Anderson, SE Barker, Y Durán, C Bartholomae, C Von Kalle, JR Heckenlively, C Kinnon, RR Ali and AJ Thrasher Nat. Med. 2006; 12 (3) :348-353

Stable rAAV-mediated transduction of rod and cone photoreceptors in the canine retina.
JWB Bainbridge, A Mistry, F Schlichtenbrede, A Smith, C Broderick, M De Alwis, A Georgiardis, PM Taylor, M Squires, C Sethi, D Charteris, AJ Thrasher, D Sargan and RR Ali Gene Ther. 2003; 10 (16): 1336-1344

In vivo gene transfer to the mouse eye using an HIV-based lentiviral vector: efficient, long-term transduction of corneal endothelium and retinal pigment epithelium.
JWB Bainbridge, C Stephens, K Parsley, C Demaison, A Halfyard, AJ Thrasher and RR Ali Gene Ther. 2001; 8 (21): 1665-1668

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