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 […]Read more...
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 […]Read more...
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 […]Read more...
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 […]Read more...
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 […]Read more...
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
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
photoreceptor ultrastructure and function in retinal degeneration slow mice by
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
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.
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
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
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
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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