Prof Robin Ali

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Personal Profile

Name: Robin Ali Email: r.ali@ucl.ac.uk
Title: Prof Tel: 020 7608 6817
Department: Inst Ophthalmology - Genetics Fax: 020 7608 6991
Position: Professor of Human Molecular Genetics Address: UCL Institute of Ophthalmology, 11-43 Bath Street, London, EC1V 9EL
Research Domain: Basic Life Sciences, Biomedical Imaging (Frontier Disciplines), Experimental Medicine, Neuroscience, Personalised Medicine Web Page: Personal Web Page

Profile

Research Description

The main focus of my research is the development of gene and stem cell therapy with the primary aim of developing novel treatments for eye disease. Over the past ten years we have been optimising gene transfer to the eye. We are engaged in a comprehensive programme of work to develop gene therapy for eye disease and in particular for disorders affecting the retina, including inherited retinal degeneration as well as complex diseases such as those associated with retinal and choroidal neovascularisation and posterior uveitis.

My research has utilised a variety of viral vectors, but my main interest is in the development of vector systems based on either adeno-associated virus (AAV) or lentiviruses. We have demonstrated the utility of these systems for gene transfer to the eye. A major aspect of our research is the development and use of a wide variety of genetic and experimental animal models of retinal disorders, including large animal models, in order to assess novel therapeutic approaches. We are engaged in a broad programme of work to demonstrate proof of concept for a number of alternative strategies, including gene replacement therapy and/or delivery of siRNA to treat animal models of inherited retinal degeneration and delivery of genes encoding angiostatic, anti-apoptotic, immunomodulatory or neurotrophic molecules to treat a variety of animal models. We are now also investigating the potential of stem or progenitor cell transplantation to repair degenerating retinae. A recent key discovery is that transplantation of rod precursor cells at a specific stage of development results in their integration and subsequent differentiation into rod photoreceptors that form synaptic connections and improve visual function in mouse models of retinal degeneration. Conversely, transplantation of progenitor or stem cells that are not at this precise ontogentic stage do not show this property and fail to integrate. We are now combining gene therapy approaches with that of stem cell transplantation and using viral vectors carrying genes encoding a variety of transcription factors in order to generate appropriate cells for transplantation from either embryonic or adult-derived stem cells.

In order to deliver new treatments, we have now established a programme of translational research. My research group includes a number of clinicians (clinical training fellows as well as senior clinican/scientists) and we have strong links with a number of biotechnology companies. We have recently established a Department of Health funded clinical trial of gene therapy for a form of severe childhood-onset retinal dystrophy due to mutations in the gene encoding RPE65. This condition is likely to be particularly amenable to effective treatment and the trial should facilitate future trials and underpin the programme of development of treatments for other retinal disorders. The first patients were enrolled in early 2007 and the first results from the trial were published in the New England Journal of Medicine in May 2008.

Research Activities

Development of novel therapies for the treatment of retinal degeneration

Differentiation of polarised epithelia

Inherited Eye Disease

Education Description

UCL Collaborators

Prof Shomi Bhattacharya; Prof Adrian Thrasher; Prof John Greenwood; Dr Virginia Calder; Prof Jane Sowden; Prof David Hunt; Prof Philip Luthert; Dr Patric Turowski; Prof Karl Matter; Prof Tony Moore; Prof Andrew Webster; Prof Maria Balda; Prof James Bainbridge

External Collaborators

Publications

    2014

    • Ali RR, Hollander AP, Kemp P, Webster A, Wilkins MR (2014). Regulating cell-based regenerative medicine: the challenges ahead.. Regen Med, 9(1), 81 - 87. doi:10.2217/rme.13.78
    • Luhmann UF, Robbie SJ, Bainbridge JW, Ali RR (2014). The relevance of chemokine signalling in modulating inherited and age-related retinal degenerations.. Adv Exp Med Biol, 801, 427 - 433. doi:10.1007/978-1-4614-3209-8_54

    2013

    • Warre-Cornish K, Barber AC, Sowden JC, Ali RR, Pearson RA (2013). Migration, integration and maturation of photoreceptor precursors following transplantation in the mouse retina.. Stem Cells Dev, , - . doi:10.1089/scd.2013.0471
    • Aguilà M, Bevilacqua D, McCulley C, Schwarz N, Athanasiou D, Kanuga N, Novoselov SS, Lange CA, Ali RR, Bainbridge JW, Gias C, Coffey PJ, Garriga P, Cheetham ME (2013). Hsp90 inhibition protects against inherited retinal degeneration.. Hum Mol Genet, , - . doi:10.1093/hmg/ddt613
    • Sundaram V, Wilde C, Aboshiha J, Cowing J, Han C, Langlo CS, Chana R, Davidson AE, Sergouniotis PI, Bainbridge JW, Ali RR, Dubra A, Rubin G, Webster AR, Moore AT, Nardini M, Carroll J, Michaelides M (2013). Retinal Structure and Function in Achromatopsia: Implications for Gene Therapy.. Ophthalmology, , - . doi:10.1016/j.ophtha.2013.08.017
    • Gonzalez-Cordero A, West EL, Pearson RA, Duran Y, Carvalho LS, Chu CJ, Naeem A, Blackford SJ, Georgiadis A, Lakowski J, Hubank M, Smith AJ, Bainbridge JW, Sowden JC, Ali RR (2013). Photoreceptor precursors derived from three-dimensional embryonic stem cell cultures integrate and mature within adult degenerate retina.. Nat Biotechnol, , - . doi:10.1038/nbt.2643
    • Mowat FM, Breuwer AR, Bartoe JT, Annear MJ, Zhang Z, Smith AJ, Bainbridge JW, Petersen-Jones SM, Ali RR (2013). RPE65 gene therapy slows cone loss in Rpe65-deficient dogs.. Gene Ther, 20(5), 545 - 555. doi:10.1038/gt.2012.63
    • Chu CJ, Herrmann P, Carvalho LS, Liyanage SE, Bainbridge JW, Ali RR, Dick AD, Luhmann UF (2013). Assessment and in vivo scoring of murine experimental autoimmune uveoretinitis using optical coherence tomography.. PLoS One, 8(5), e63002 - . doi:10.1371/journal.pone.0063002
    • Luhmann UF, Carvalho LS, Robbie SJ, Bainbridge JW, Ali RR (2013). Reply to comment on "Ccl2, Cx3cr1 and Ccl2/Cx3cr1 chemokine deficiencies are not sufficient to cause age-related retinal degeneration" by Luhmann et al. (Exp. Eye Res. 107, February 2013, 80-87).. Exp Eye Res, 111, 136 - . doi:10.1016/j.exer.2013.02.001
    • Luhmann UF, Carvalho LS, Robbie SJ, Cowing JA, Duran Y, Munro PM, Bainbridge JW, Ali RR (2013). Ccl2, Cx3cr1 and Ccl2/Cx3cr1 chemokine deficiencies are not sufficient to cause age-related retinal degeneration.. Exp Eye Res, 107, 80 - 87. doi:10.1016/j.exer.2012.11.015
    • Boucherie C, Mukherjee S, Henckaerts E, Thrasher AJ, Sowden JC, Ali RR (2013). Brief report: self-organizing neuroepithelium from human pluripotent stem cells facilitates derivation of photoreceptors.. Stem Cells, 31(2), 408 - 414. doi:10.1002/stem.1268

    2012

    • West EL, Pearson RA, Duran Y, Gonzalez-Cordero A, MacLaren RE, Smith AJ, Sowden JC, Ali RR (2012). Manipulation of the recipient retinal environment by ectopic expression of neurotrophic growth factors can improve transplanted photoreceptor integration and survival.. Cell Transplant, 21(5), 871 - 887. doi:10.3727/096368911X623871
    • Kampik D, Ali RR, Larkin DF (2012). Experimental gene transfer to the corneal endothelium.. Exp Eye Res, 95(1), 54 - 59. doi:10.1016/j.exer.2011.07.001
    • Lange CA, Luhmann UF, Mowat FM, Georgiadis A, West EL, Abrahams S, Sayed H, Powner MB, Fruttiger M, Smith AJ, Sowden JC, Maxwell PH, Ali RR, Bainbridge JW (2012). Von Hippel-Lindau protein in the RPE is essential for normal ocular growth and vascular development.. Development, 139(13), 2340 - 2350. doi:10.1242/dev.070813
    • West EL, Gonzalez-Cordero A, Hippert C, Osakada F, Martinez-Barbera JP, Pearson RA, Sowden JC, Takahashi M, Ali RR (2012). Defining the integration capacity of embryonic stem cell-derived photoreceptor precursors.. Stem Cells, 30(7), 1424 - 1435. doi:10.1002/stem.1123
    • 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 (2012). Restoration of vision after transplantation of photoreceptors.. Nature, 485(7396), 99 - 103. doi:10.1038/nature10997
    • Tan MH, Mackay DS, Cowing J, Tran HV, Smith AJ, Wright GA, Dev-Borman A, Henderson RH, Moradi P, Russell-Eggitt I, MacLaren RE, Robson AG, Cheetham ME, Thompson DA, Webster AR, Michaelides M, Ali RR, Moore AT (2012). Leber congenital amaurosis associated with AIPL1: challenges in ascribing disease causation, clinical findings, and implications for gene therapy.. PLoS One, 7(3), e32330 - . doi:10.1371/journal.pone.0032330
    • Petsoglou C, Balaggan KS, Dart JKG, Bunce C, Xing W, Ali RR, Tuft SJ (2012). Subconjunctival bevacizumab induces regression of corneal neovascularisation: A pilot randomised placebo-controlled double-masked trial. British Journal of Ophthalmology, , - .
    • Luhmann UF, Lange CA, Robbie S, Munro PM, Cowing JA, Armer HE, Luong V, Carvalho LS, MacLaren RE, Fitzke FW, Bainbridge JW, Ali RR (2012). Differential modulation of retinal degeneration by Ccl2 and Cx3cr1 chemokine signalling.. PLoS One, 7(4), e35551 - . doi:10.1371/journal.pone.0035551
    • Ali RR (2012). Gene therapy for retinal dystrophies: twenty years in the making.. Hum Gene Ther, 23(4), 337 - 339. doi:10.1089/hum.2012.2506
    • Mowat FM, Gonzalez F, Luhmann UF, Lange CA, Duran Y, Smith AJ, Maxwell PH, Ali RR, Bainbridge JW (2012). Endogenous erythropoietin protects neuroretinal function in ischemic retinopathy.. Am J Pathol, 180(4), 1726 - 1739. doi:10.1016/j.ajpath.2011.12.033
    • Ali RR (2012). Ocular gene therapy: introduction to the special issue.. Gene Ther, 19(2), 119 - 120. doi:10.1038/gt.2011.189
    • Luhmann UF, Ali RR (2012). Local vs. systemic mononuclear phagocytes in age-related macular degeneration and their regulation by CCL2-CCR2 and CX3CL1-CX3CR1 chemokine signalling.. Adv Exp Med Biol, 723, 17 - 22. doi:10.1007/978-1-4614-0631-0_3
    • Sundaram V, Moore AT, Ali RR, Bainbridge JW (2012). Retinal dystrophies and gene therapy.. Eur J Pediatr, 171(5), 757 - 765. doi:10.1007/s00431-011-1615-2
    • Smith AJ, Bainbridge JW, Ali RR (2012). Gene supplementation therapy for recessive forms of inherited retinal dystrophies.. Gene Ther, 19(2), 154 - 161. doi:10.1038/gt.2011.161
    • Sundaram V, Wilde C, Webster AR, Moore AT, Nardini M, Ali RR, Bainbridge JW, Carroll J, Michaelides M (2012). Cone Photoreceptor Structure in Achromatopsia.

    2011

    • West EL, Gonzalez-Cordero A, Hippert C, Duran Y, Pearson RA, Sowden JC, Ali RR (2011). Embryonic stem cell-derived retinal cells transplanted to the adult retina.
    • Bartholomae CC, Arens A, Balaggan KS, Yáñez-Muñoz RJ, Montini E, Howe SJ, Paruzynski A, Korn B, Appelt JU, Macneil A, Cesana D, Abel U, Glimm H, Naldini L, Ali RR, Thrasher AJ, von Kalle C, Schmidt M (2011). Lentiviral vector integration profiles differ in rodent postmitotic tissues.. Mol Ther, 19(4), 703 - 710. doi:10.1038/mt.2011.19
    • Gualdoni S, Baron M, Lakowski J, Decembrini S, Pearson RA, Ali RR, Sowden JC (2011). Isolation and culture of adult ciliary epithelial cells, previously identified as retinal stem cells, and retinal progenitor cells.. Curr Protoc Stem Cell Biol, Chapter 1, Unit - 1H.4. doi:10.1002/9780470151808.sc01h04s19
    • Lakowski J, Han YT, Pearson RA, Gonzalez-Cordero A, West EL, Gualdoni S, Barber AC, Hubank M, Ali RR, Sowden JC (2011). Effective transplantation of photoreceptor precursor cells selected via cell surface antigen expression.. Stem Cells, 29(9), 1391 - 1404. doi:10.1002/stem.694
    • Boucherie C, Sowden JC, Ali RR (2011). Induced pluripotent stem cell technology for generating photoreceptors.. Regen Med, 6(4), 469 - 479. doi:10.2217/rme.11.37
    • Ali RR, Sowden JC (2011). Regenerative medicine: DIY eye.. Nature, 472(7341), 42 - 43. doi:10.1038/472042a
    • Buch PK, Mihelec M, Cottrill P, Wilkie SE, Pearson RA, Duran Y, West EL, Michaelides M, Ali RR, Hunt DM (2011). Dominant cone-rod dystrophy: a mouse model generated by gene targeting of the GCAP1/Guca1a gene.. PLoS One, 6(3), e18089 - . doi:10.1371/journal.pone.0018089
    • Annear MJ, Bartoe JT, Barker SE, Smith AJ, Curran PG, Bainbridge JW, Ali RR, Petersen-Jones SM (2011). Gene therapy in the second eye of RPE65-deficient dogs improves retinal function. GENE THER, 18(1), 53 - 61. doi:10.1038/gt.2010.111
    • Decembrini S, Cananzi M, Gualdoni S, Battersby A, Allen N, Pearson RA, Ali RR, De Coppi P, Sowden JC (2011). Comparative analysis of the retinal potential of embryonic stem cells and amniotic fluid-derived stem cells.. Stem Cells Dev, 20(5), 851 - 863. doi:10.1089/scd.2010.0291
    • Luhmann UFO, Kampik D, Basche M, Smith AJ, Larkin DFP, Ali RR (2011). Replication of retinal pigment epithelium can be induced by non integrating lentiviral gene transfer of E2F2. HUM GENE THER, 22(10), A78 - A78.
    • Carvalho LS, Mihelec M, Pearson RA, Bainbridge JWB, Smith AJ, Ali RR (2011). Gene replacement therapy in mouse models of inherited cone photoreceptor disorders. HUM GENE THER, 22(10), A25 - A25.
    • Mihelec M, Pearson RA, Robbie SJ, Buch PK, Azam SA, Bainbridge JW, Smith AJ, Ali RR (2011). 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.. Hum Gene Ther, 22(10), 1179 - 1190. doi:10.1089/hum.2011.069
    • Ali RR (2011). Development of stem cell therapy for the treatment of retinal degeneration. HUM GENE THER, 22(10), A10 - A11.
    • Lange CA, Stavrakas P, Luhmann UF, de Silva DJ, Ali RR, Gregor ZJ, Bainbridge JW (2011). Intraocular oxygen distribution in advanced proliferative diabetic retinopathy.. Am J Ophthalmol, 152(3), 406 - 412.e3. doi:10.1016/j.ajo.2011.02.014
    • Carvalho LS, Xu J, Pearson RA, Smith AJ, Bainbridge JW, Morris LM, Fliesler SJ, Ding XQ, Ali RR (2011). Long-term and age-dependent restoration of visual function in a mouse model of CNGB3-associated achromatopsia following gene therapy.. Hum Mol Genet, 20(16), 3161 - 3175. doi:10.1093/hmg/ddr218
    • Hippert C, Barber AC, Georgiadis A, Smith AJ, Swoden JC, Ali RR, Pearson RA (2011). Using shGFAP to examine the impact of gliosis on photoreceptor transplantation efficacy.

    2010

    • Ali RR, Bainbridge JW, Smith AJ, Robbie S, Fitzke FW, Holder GE, Stockman A, Yzer Z, Van den Born I, Rubin GS (2010). Clinical trial of gene therapy for early onset severe retinal dystrophy resulting from defects in RPE65.
    • Georgiadis A, Tschernutter M, Bainbridge JW, Robbie SJ, McIntosh J, Nathwani AC, Smith AJ, Ali RR (2010). AAV-mediated knockdown of peripherin-2 in vivo using miRNA-based hairpins.. Gene Ther, 17(4), 486 - 493. doi:10.1038/gt.2009.162
    • Ali RR, Bainbridge JWB, Smith AJ, Fitzke FW, Holder GE, Stockman A, Bhattacharya SS, Rubin GS, Yzer S, van den Born I, Moore AT (2010). Clinical Trial of Gene Therapy for Early Onset Severe Retinal Dystrophy Resulting from Defects in RPE65.
    • Lakowski J, Baron M, Bainbridge J, Barber AC, Pearson RA, Ali RR, Sowden JC (2010). Cone and rod photoreceptor transplantation in models of the childhood retinopathy Leber congenital amaurosis using flow-sorted Crx-positive donor cells. Hum.Mol.Genet, 19, 4545 - 4559.
    • West EL, Pearson RA, Barker SE, Luhmann UF, Maclaren RE, Barber AC, Duran Y, Smith AJ, Sowden JC, Ali RR (2010). Long-term survival of photoreceptors transplanted into the adult murine neural retina requires immune modulation.. Stem Cells, 28(11), 1997 - 2007. doi:10.1002/stem.520
    • Pearson RA, Barber AC, West EL, MacLaren RE, Duran Y, Bainbridge JW, Sowden JC, Ali RR (2010). Targeted disruption of outer limiting membrane junctional proteins (Crb1 and ZO-1) increases integration of transplanted photoreceptor precursors into the adult wild-type and degenerating retina.. Cell Transplant, 19(4), 487 - 503. doi:10.3727/096368909X486057
    • West EL, Gonzalez CA, Pearson RA, Barber A, Sowden JC, Ali RR (2010). Immune Responses Affect the Survival of Integrated Photoreceptor Precursors Transplanted to the Adult Murine Retina.
    • Barber A, Bainbridge JW, Sowden J, Ali RR, Pearson RA (2010). Gliosis and Extracellular Matrix Proteins: Their Role in Rod Photoreceptor Transplantation in the Degenerating Retina.
    • Sun X, Pawlyk B, Xu X, Liu X, Bulgakov OV, Adamian M, Sandberg MA, Khani SC, Tan MH, Smith AJ, Ali RR, Li T (2010). Gene therapy with a promoter targeting both rods and cones rescues retinal degeneration caused by AIPL1 mutations. GENE THER, 17(1), 117 - 131. doi:10.1038/gt.2009.104
    • Gualdoni S, Baron M, Lakowski J, Decembrini S, Smith AJ, Pearson RA, Ali RR, Sowden JC (2010). Adult ciliary epithelial cells, previously identified as retinal stem cells with potential for retinal repair, fail to differentiate into new rod photoreceptors.. Stem Cells, 28(6), 1048 - 1059. doi:10.1002/stem.423
    • Kampik D, Smith A, Luhmann UFO, Buch P, Larkin DFP, Ali RR (2010). Non-Integrating Lentiviral Gene Transfer of E2F2 to Induce Corneal Endothelial Cell Replication.
    • Aartsen WM, van Cleef KW, Pellissier LP, Hoek RM, Vos RM, Blits B, Ehlert EM, Balaggan KS, Ali RR, Verhaagen J, Wijnholds J (2010). GFAP-driven GFP expression in activated mouse Müller glial cells aligning retinal blood vessels following intravitreal injection of AAV2/6 vectors.. PLoS One, 5(8), e12387 - . doi:10.1371/journal.pone.0012387
    • Smith AJ, Bainbridge JWB, Robbie S, Barker SE, Rubin GS, Moore AT, Ali RR (2010). Improved Retinal Function After AAV Gene Therapy: An Update on the RPE65 Clinical Trial.
    • Mowat FM, Luhmann UF, Smith AJ, Lange C, Duran Y, Harten S, Shukla D, Maxwell PH, Ali RR, Bainbridge JW (2010). HIF-1alpha and HIF-2alpha are differentially activated in distinct cell populations in retinal ischaemia.. PLoS One, 5(6), e11103 - . doi:10.1371/journal.pone.0011103
    • Georgiadis A, Tschernutter M, Bainbridge JW, Balaggan KS, Mowat F, West EL, Munro PM, Thrasher AJ, Matter K, Balda MS, Ali RR (2010). The tight junction associated signalling proteins ZO-1 and ZONAB regulate retinal pigment epithelium homeostasis in mice.. PLoS One, 5(12), e15730 - . doi:10.1371/journal.pone.0015730

    2009

    • Barber AC, Bainbridge JWB, Sowden JC, Ali RR, Pearson RA (2009). The role of gliosis and extracellular matrix proteins in rod photoreceptor precursor transplantation in the degenerating retina.
    • Smith AJ, Bainbridge JW, Ali RR (2009). Prospects for retinal gene replacement therapy. Trends Genet., 25(4), 156 - 165.
    • Ali RR (2009). Gene Therapy for Inherited Retinal Degeneration: From Mouse to Man.
    • West EL, Pearson RA, MacLaren RE, Sowden JC, Ali RR (2009). Cell transplantation strategies for retinal repair. Prog.Brain Res., 175, 3 - 21.
    • Gabriel R, Eckenberg R, Paruzynski A, Bartholomae CC, Nowrouzi A, Arens A, Howe SJ, Recchia A, Cattoglio C, Wang W, Faber K, Schwarzwaelder K, Kirsten R, Deichmann A, Ball CR, Balaggan KS, Yanez-Munoz RJ, Ali RR, Gaspar HB, Biasco L, Aiuti A, Cesana D, Montini E, Naldini L, Cohen-Haguenauer O, Mavilio F, Thrasher AJ, Glimm H, von Kalle C, Saurin W, Schmidt M (2009). Comprehensive genomic access to vector integration in clinical gene therapy. Nature Medicine, 15(12), 1431 - 1U13.
    • Luhmann UF, Robbie S, Munro PM, Barker SE, Duran Y, Luong V, Fitzke FW, Bainbridge J, Ali RR, Maclaren R (2009). The drusen-like phenotype in aging Ccl2 knockout mice is caused by an accelerated accumulation of swollen autofluorescent subretinal macrophages. Invest Ophthalmol.Vis.Sci., , - .
    • Barker SE, Broderick CA, Robbie SJ, Duran Y, Natkunarajah M, Buch P, Balaggan KS, Maclaren RE, Bainbridge JW, Smith AJ, Ali RR (2009). Subretinal delivery of adeno-associated virus serotype 2 results in minimal immune responses that allow repeat vector administration in immunocompetent mice. J.Gene Med., 11(6), 486 - 497. doi:10.1002/jgm.1327
    • Tan MH, Smith AJ, Pawlyk B, Xu X, Liu X, Bainbridge JB, Basche M, McIntosh J, Tran HV, Nathwani A, Li T, Ali RR (2009). 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 AAV2/2 and AAV2/8 vectors. Hum.Mol.Genet., 18(12), 2099 - 2114.
    • Paruzynski A, Gabriel R, Eckenberg R, Bartholomae CC, Nowrouzi A, Arens A, Howe SJ, Recchia A, Cattoglio C, Wang W, Schwarzwaelder K, Kirsten R, Deichmann A, Ball CR, Balaggan KS, Yanez-Munoz RJ, Ali RR, Gaspar HB, Biasco L, Aiuti A, Cesana D, Montini E, Naldini L, Cohen-Haguenauer O, Mavilio F, Thrasher AJ, Glimm H, von Kalle C, Saurin W, Schmidt M (2009). Modeling and Increasing the Genome Accessibility of Viral Integration Sites.

    2008

    • Buch PK, McIntosh J, Georgiadis A, Mowat F, Tschernutter M, Smith AJ, Nathwani AC, Ali RR (2008). Pseudotyped adeno-associated viral vectors to enhance gene replacement therapy in a mouse model of retinal degeneration.
    • Buch PK, Bainbridge JW, Ali RR (2008). AAV-mediated gene therapy for retinal disorders: from mouse to man. Gene Ther., 15(11), 849 - 857.
    • West EL, Pearson RA, Duran Y, Luhmann UFO, Barker SE, Smith AJ, Sowden JC, MacLaren RE, Ali RR (2008). Overexpression of developmentally regulated neurotrophins in the adult mouse retina to facilitate integration of transplanted photoreceptor precursor cells.
    • Kokkinopoulos I, Pearson RA, Macneil A, Dhomen NS, Maclaren RE, Ali RR, Sowden JC (2008). Isolation and characterisation of neural progenitor cells from the adult Chx10(orJ/orJ) central neural retina. Molecular and Cellular Neuroscience, 38(3), 359 - 373. doi:10.1016/j.mcn.2008.03.008
    • Bainbridge JW, Smith AJ, Barker SS, Robbie S, Henderson R, Balaggan K, Viswanathan A, Holder GE, Stockman A, Tyler N, Petersen-Jones S, Bhattacharya SS, Thrasher AJ, Fitzke FW, Carter BJ, Rubin GS, Moore AT, Ali RR (2008). Effect of gene therapy on visual function in Leber\'s congenital amaurosis. N.Engl.J.Med., 358(21), 2231 - 2239.
    • Barker SE, Smith AJ, Luhmann UFO, Bainbridge JW, MacLaren RE, Ali RR (2008). Development of a murine model of age-related macular degeneration by overexpression of complement component 3 in the RPE of MCP-1(-/-) and wild-type mice.
    • Natkunarajah M, Trittibach P, McIntosh J, Duran Y, Barker SE, Smith AJ, Nathwani AC, Ali RR (2008). Assessment of ocular transduction using single-stranded and self-complementary recombinant adeno-associated virus serotype 2/8. Gene Therapy, 15(6), 463 - 467.
    • Mowat FM, Petersen-Jones SM, Williamson H, Williams DL, Luthert PJ, Ali RR, Bainbridge JW (2008). Topographical characterization of cone photoreceptors and the area centralis of the canine retina. Mol Vis., 14, 2518 - 2527.
    • Bainbridge JW, Ali RR (2008). Success in sight: The eyes have it! Ocular gene therapy trials for LCA look promising. Gene Ther., 15(17), 1191 - 1192.
    • Bartholomae CC, Yanez-Munoz RJ, Balaggan KS, Howe SJ, MacNeil A, Ali RR, Thrasher AJ, von Kalle C, Schmidt M (2008). Nearly random integration site distribution in postmitotic rodent tissues transduced with lentiviral SIN-vectors. Blood Cells, Molecules, and Diseases, 40(2), 6 - .
    • Bartholomae CC, Yanez-Munoz RJ, Balaggan KS, Howe SJ, MacNeil A, Ali RR, Thrasher AJ, von Kalle C, Schmidt M (2008). Nearly random integration site distribution in postmitotic rodent tissues transduced with lentiviral SIN-vectors. doi:10.1016/j.bcmd.2007.10.016
    • Trittibach P, Barker SE, Broderick CA, Natkunarajah M, Duran Y, Robbie SJ, Bainbridge JWB, Smith AJ, Sarra G-M, Dick AD, Ali RR (2008). Lentiviral-vector-mediated expression of murine IL-1 receptor antagonist or IL-10 reduces the severity of endotoxin-induced uveitis. Gene Therapy, 15(22), 1478 - 1488. doi:10.1038/gt.2008.109
    • Bainbridge JW, Ali RR (2008). Keeping an eye on clinical trials in 2008. Gene Ther., 15(9), 633 - 634.
    • Gabriel R, Paruzynski A, Eckenberg R, Bartholomae CC, Nowrouzi A, Wang W, Schwarzwaelder K, Arens A, Kirsten R, Deichmann A, Ball CR, Howe SJ, Recchia A, Claudia C, Balaggan KS, Yanez-Munoz RJ, Ali RR, Mavilio F, Gaspar HB, Thrasher AJ, Glimm H, von Kalle C, Saurin W, Schmidt M (2008). Assessing the genome accessibility of viral integration sites. Human Gene Therapy, 19(10), 1085 - 1085.
    • Gabriel R, Paruzynski A, Eckenberg R, Bartholomae CC, Nowrouzi A, Wang W, Schwarzwaelder K, Arens A, Kirsten R, Deichmann A, Ball C, Howe SJ, Recchia A, Cattoglio C, Balaggan KS, Yanez-Munoz RJ, Ali RR, Mavilio F, Gaspar B, Thrasher A, Glimm H, von Kalle C, Saurin W, Schmidt M (2008). Comprehensive and Unbiased Integration Site Analysis in Clinical Gene Therapy.
    • West EL, Pearson RA, Tschernutter M, Sowden JC, Maclaren RE, Ali RR (2008). Pharmacological disruption of the outer limiting membrane leads to increased retinal integration of transplanted photoreceptor precursors. Exp.Eye Res., 86(4), 601 - 611.

    2007

    • Alexander BL, Ali RR, Alton EWFW, Bainbridge JW, Braun S, Cheng SH, Flotte TR, Gaspar HB, Grez M, Griesenbach U, Kaplitt MG, Ott MG, Seger R, Simons M, Thrasher AJ, Thrasher AZ, Herttuala SY (2007). Progress and prospects: gene therapy clinical trials (part 1) (vol 14, pg 1439, 2007). GENE THER, 14(24), 1754 - 1754. doi:10.1038/sj.gt.3303075
    • Buch PK, Maclaren RE, Ali RR (2007). Neuroprotective gene therapy for the treatment of inherited retinal degeneration. Curr.Gene Ther., 7(6), 434 - 445.
    • Ali RR, Pearson RA, MacLaren RE, West E, MacNeil A, Douglas RH, Salt TE, Akimoto M, Swaroop A, Sowden JC (2007). Kinetics of Rod Photoreceptor Precursor Integration Following Transplantation in the Adult Mouse Retina.
    • MacNeil A, Pearson RA, MacLaren RE, Smith AJ, Sowden JC, Ali RR (2007). Comparative analysis of progenitor cells isolated from the iris, pars plana, and ciliary body of the adult porcine eye. Stem Cells, 25(10), 2430 - 2438. doi:10.1634/stemcells.2007-0035
    • MacNeil A, Pearson RA, MacLaren RE, Buch PK, Smith AJ, Sowden JC, Ali RR (2007). Comparative Analysis of Neural Stem Cells Isolated From the Iris, Pars Plana and Ciliary Body of the Porcine Eye.
    • Khani SC, Pawlyk BS, Bulgakov OV, Kasperek E, Young JE, Adamian M, Sun X, Smith AJ, Ali RR, Li T (2007). AAV-mediated expression targeting of rod and cone photoreceptors with a human rhodopsin kinase promoter. Invest Ophthalmol Vis.Sci., 48(9), 3954 - 3961.
    • Le Meur G, Stieger K, Smith AJ, Weber M, Deschamps JY, Nivard D, Mendes-Madeira A, Provost N, Pereon Y, Cherel Y, Ali RR, Hamel C, Moullier P, Rolling F (2007). Restoration of vision in RPE65-deficient Briard dogs using an AAV serotype 4 vector that specifically targets the retinal pigmented epithelium. Gene Ther., 14(4), 292 - 303.
    • Alexander BL, Ali RR, Alton EW, Bainbridge JW, Braun S, Cheng SH, Flotte TR, Gaspar HB, Grez M, Griesenbach U, Kaplitt MG, Ott MG, Seger R, Simons M, Thrasher AJ, Thrasher AZ, Yla-Herttuala S (2007). Progress and prospects: gene therapy clinical trials (part 1). Gene Ther., 14(20), 1439 - 1447.
    • Yokoi K, Zhang HS, Kachi S, Balaggan KS, Yu Q, Guschin D, Kunis M, Surosky R, Africa LM, Bainbridge JW, Spratt SK, Gregory PD, Ali RR, Campochiaro PA (2007). Gene transfer of an engineered zinc finger protein enhances the anti-angiogenic defense system. Mol Ther., 15(11), 1917 - 1923.
    • Yokoi K, Zhang HS, Kachi S, Balaggan KS, Yu Q, Guschin D, Kunis M, Surosky R, Africa LM, Bainbridge JW, Spratt SK, Gregory PD, Ali RR, Campochiaro PA (2007). Gene transfer of an engineered zinc finger protein enhances the anti-angiogenic Defense system. MOL THER, 15(11), 1917 - 1923. doi:10.1038/sj.mt.6300280
    • Maclaren RE, Uppal GS, Balaggan KS, Tufail A, Munro PM, Milliken AB, Ali RR, Rubin GS, Aylward GW, da Cruz L (2007). Autologous transplantation of the retinal pigment epithelium and choroid in the treatment of neovascular age-related macular degeneration. Ophthalmology, 114(3), 561 - 570. doi:10.1016/j.ophtha.2006.06.049
    • West EL, Pearson RA, Sowden JC, MacLaren RE, Ali RR (2007). The Outer Limiting Membrane as a Barrier to Photoreceptor Integration, After Transplantation to the Subretinal Space of the Adult Mouse.

    2006

    • Ali RR, MacLaren RE, Pearson RA, MacNeil A, Sowden JC (2006). Successful Transplantation of Photoreceptors into the Retina of an Adult Mouse Model of Human Retinitis Pigmentosa.
    • MacLaren RE, Pearson RA, MacNeil A, Douglas RH, Salt TE, Akimoto M, Swaroop A, Sowden JC, Ali RR (2006). Retinal repair by transplantation of photoreceptor precursors.. Nature, 444(7116), 203 - 207. doi:10.1038/nature05161
    • Sourisseau T, Georgiadis A, Tsapara A, Ali RR, Pestell R, Matter K, Balda MS (2006). Regulation of PCNA and cyclin D1 expression and epithelial morphogenesis by the ZO-1 regulated transcription factor ZONAB/DbpA. Molecular and Cellular Biology, 26(6), 2387 - 2398. doi:10.1128/MCB.26.6.2387-2398.2006
    • Bigger BW, Siapati EK, Mistry A, Waddington SN, Nivsarkar MS, Jacobs L, Perrett R, Holder MV, Ridler C, Kemball-Cook G, Ali RR, Forbes SJ, Coutelle C, Wright N, Alison M, Thrasher AJ, Bonnet D, Themis M (2006). Permanent partial phenotypic correction and tolerance in a mouse model of hemophilia B by stem cell gene delivery of human factor IX. Gene Therapy, 13(2), 117 - 126.
    • Tschernutter M, Jenkins SA, Waseem NH, Saihan Z, Holder GE, Bird AC, Bhattacharya SS, Ali RR, Webster AR (2006). Clinical characterisation of a family with retinal dystrophy caused by mutation in the Mertk gene. British Journal of Ophthalmology, 90(6), 718 - 723.
    • Dhomen NS, Balaggan KS, Pearson RA, Bainbridge JW, Levine EM, Ali RR, Sowden JC (2006). Absence of Chx10 causes neural progenitors to persist in the adult retina. Investigative Ophthalmology and Visual Science, 47(1), 386 - 396. doi:10.1167/iovs.05-0428
    • Balaggan KS, Binley K, Esapa M, Iqball S, Askham Z, Kan O, Tschernutter M, Bainbridge JW, Naylor S, Ali RR (2006). Stable and efficient intraocular gene transfer using pseudotyped EIAV lentiviral vectors. The Journal of Gene Medicine, 8(3), 275 - 285.
    • Broderick CA, Smith AJ, Balaggan KS, Georgiadis A, Buch PK, Trittibach PC, Barker SE, Sarra GM, Thrasher AJ, Dick AD, Ali RR (2006). Local administration of an adeno-associated viral vector expressing IL-10 reduces monocyte infiltration and subsequent photoreceptor damage during experimental autoimmune uveitis (vol 12, pg 369, 2005). Molecular Therapy, 13(4), 829 - 829.
    • Surace EM, Balaggan KS, Tessitore A, Mussolino C, Cotugno G, Bonetti C, Vitale A, Ali RR, Auricchio A (2006). Inhibition of ocular neovascularization by hedgehog blockade. Molecular Therapy, 13(3), 573 - 579.
    • Buch PK, MacLaren RE, Duran Y, Balaggan KS, MacNeil A, Schlichtenbrede FC, Smith AJ, Ali RR (2006). In contrast to AAV-mediated Cntf expression, AAV-mediated Gdnf expression enhances gene replacement therapy in rodent models of retinal degeneration. Molecular Therapy, 14(5), 700 - 709. doi:10.1016/j.ymthe.2006.05.019
    • Bainbridge JW, Tan MH, Ali RR (2006). Gene therapy progress and prospects: the eye. Gene Therapy, 13(16), 1191 - 1197.
    • Rolling F, Le Meur G, Stieger K, Smith AJ, Weber M, Deschamps JY, Nivard D, Mendes-Madeira A, Provost N, Pereon Y, Cherel Y, Ali RR, Hamel C, Moullier P, Rolling F (2006). Gene therapeutic prospects in early onset of severe retinal dystrophy: restoration of vision in RPE65 Briard dogs using an AAV serotype 4 vector that specifically targets the retinal pigmented epithelium. Bulletin et Mémoires de l'Académie Royale de Médecine de Belgique, 161(10-12), 497 - 508.
    • Balaggan KS, Binley K, Esapa M, MacLaren RE, Iqball S, Duran Y, Pearson RA, Kan O, Barker SE, Smith AJ, Bainbridge JW, Naylor S, Ali RR (2006). EIAV vector-mediated delivery of endostatin or angiostatin inhibits angiogenesis and vascular hyperpermeability in experimental CNV. Gene therapy, 13(15), 1153 - 1165. doi:10.1038/sj.gt.3302769
    • Yanez-Munoz RJ, Balaggan KS, MacNeil A, Howe SJ, Schmidt M, Smith AJ, Buch P, MacLaren RE, Anderson PN, Barker SE, Duran Y, Bartholomae C, Von Kalle C, Heckenlively JR, Kinnon C, Ali RR, Thrasher AJ (2006). Effective gene therapy with non-integrating lentiviral vectors. Nature Medicine, 12(3), 348 - 353. doi:10.1038/nm1365
    • MacLaren RE, Buch PK, Smith AJ, Balaggan KS, MacNeil A, Taylor JS, Osborne NN, Ali RR (2006). CNTF gene transfer protects ganglion cells in rat retinae undergoing focal injury and branch vessel occlusion. Experimental Eye Research, 83(5), 1118 - 1127. doi:10.1016/j.exer.2006.05.019

    2005

    • Pawlyk BS, Smith AJ, Buch PK, Adamian M, Hong DH, Sandberg MA, Ali RR, Li T (2005). Gene replacement therapy rescues photoreceptor degeneration in a murine model of Leber congenital amaurosis lacking RPGRIP. INVEST OPHTHALMOL VIS SCI, 46(9), 3039 - 3045.
    • McAlister JC, Joyce NC, Harris DL, Ali RR, Larkin DF (2005). Induction of replication in human corneal endothelial cells by E2F2 transcription factor cDNA transfer. INVEST OPHTHALMOL VIS SCI, 46(10), 3597 - 3603.
    • Surace EM, Balaggan KS, Tessitore A, Mussolino C, Cotugno G, Bonetti C, Vitale A, Ali RR, Auricchio A (2005). Inhibition of Ocular Neovascularization by Hedgehog Blockade. MOL THER, , - .
    • Broderick CA, Smith AJ, Balaggan KS, Georgarias A, Buch PK, Trittibach PC, Barker SE, Sarra GM, Thrasher AJ, Dick AD, Ali RR (2005). Local administration of an adeno-associated viral vector expressing IL-10 reduces monocyte infiltration and subsequent photoreceptor damage during experimental autoimmune uveitis. MOL THER, 12(2), 369 - 373.
    • Tschernutter M, Schlichtenbrede FC, Howe S, Balaggan KS, Munro PM, Bainbridge JW, Thrasher AJ, Smith AJ, Ali RR (2005). Long-term preservation of retinal function in the RCS rat model of retinitis pigmentosa following lentivirus-mediated gene therapy. GENE THER, 12(8), 694 - 701.
    • Guo L, Moss SE, Alexander RA, Ali R, Fitzke F, Cordeiro MF (2005). Retinal ganglion cell apoptosis in glaucoma is related to intraocular pressure and IOP-induced effects on extracellular matrix. Investigative Ophthalmology and Visual Science, 46(1), 175 - 182. doi:10.1167/iovs.04-0832
    • MacLaren RE, Ali RR, Thrasher AJ (2005). Risks of gene therapy should be weighed against lack of alternatives for many diseases. BMJ (Clinical Research Ed.), 330(7494), 791 - . doi:10.1136/bmj.330.7494.791-a
    • Balaggan KS, Binley K, Esapa M, Iqball S, Askham Z, Kan O, Tschernutter M, Bainbridge JW, Naylor S, Ali RR (2005). Stable and efficient intraocular gene transfer using pseudotyped EIAV lentiviral vectors. J GENE MED, , - .
    • Yanez RJ, Balaggan KS, Smith AJ, Buch P, Howe S, MacNeilA D, Y A, P N K, C A, R R T, A J (2005). Effective Gene Therapy with Non-Integrating Lentiviral Vectors.
    • MacLaren RE, MacNeil A, Pearson RA, Sowden JC, Ali RR (2005). Immature Neural Retinal Cells Integrate and Differentiate Into Photoreceptors When Transplanted Into the Mouse Subreitnal Space at Post-natal Day 1.
    • Ali R, Aller M, Luscher B, Brickley SG, Korpi ER, Cull-Candy SG, Wisden W, Farrant M (2005). Loss of inhibitory postsynaptic currents following selective ablation of the GABAA receptor γ2 subunit in cerebellar granule cells of mice..
    • Sethi CSS, F Lewis GPF, S K Luthert PJC, D G Ali RR (2005). Subretinal CNTF gene delivery in a feline model: Cell transfection and morphological sequelae. Investigative Ophthalmology and Visual Science, 46, - .
    • Parsley KL, Gaspar HB, Howe S, King D, Gilmour KG, Zhang F, Sinclair J, Von Kalle C, Ali RR, Kinnon C, Thrasher AJ (2005). Update on treatment of human SCID-X1 by somatic gene therapy using a pseudotyped gammaretroviral vector.
    • Sarra GM, Sarra FG, Schlichtenbrede FC, Trittibach P, Estermann S, Tsiroukis E, Ali RR, Luthert PJ, Reichel MB (2005). Effect of steroidal and non-steroidal drugs on the microglia activation pattern and the course of degeneration in the retinal degeneration slow mouse. OPHTHALMIC RES, 37(2), 72 - 82.

    2004

    • Schlichtenbrede FC, Smith AJ, Bainbridge JW, Thrasher AJ, Salt TE, Ali RR (2004). Improvement of neuronal visual responses in the superior colliculus in Prph2(Rd2/Rd2) mice following gene therapy.. Molecular and Cellular Neuroscience, 25(1), 103 - 110. doi:10.1016/j.mcn.2003.09.020
    • Gaspar HB, Parsley KL, Howe S, King D, Gilmour KC, Sinclair J, Brouns G, Schmidt M, Von Kalle C, Barington T, Jakobsen MA, Christensen HO, Al Ghonaium A, White HN, Smith JL, Levinsky RJ, Ali RR, Kinnon C, Thrasher AJ (2004). Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector. The Lancet, 364(9452), 2181 - 2187. doi:10.1016/S0140-6736(04)17590-9
    • Balaggan KS, Mistry A, Georgiadis T, Broderick C, Bainbridge J, Thrasher AJ, Ali RR (2004). Inhibition of experimental choroidal neovascularisation by lentivirus mediated delivery of pigment epithelium derived factor. Investigative Ophthalmology and Visual Science, 45, 3705 - .
    • Dhomen NS, Balaggan KS, Bainbridge JW, Ali RR, Sowden JC (2004). Chx10 as a regulator of retinal stem cells..
    • Rota R, Riccioni T, Zaccarini M, Lamartina S, Gallo AD, Fusco A, Kovesdi I, Balestrazzi E, Abeni DC, Ali RR, Capogrossi MC (2004). Marked inhibition of retinal neovascularization in rats following soluble-flt-1 gene transfer.. The Journal of Gene Medicine, 6(9), 992 - 1002.
    • Hughes EH, Schlichtenbrede FC, Murphy CC, Broderick C, van Rooijen N, Ali RR, Dick AD (2004). Minocycline delays photoreceptor death in the rds mouse through a microglia-independent mechanism.. Experimental Eye Research, 78(6), 1077 - 1084.
    • Waddington SN, Nivsarkar M, Mistry A, Buckley SMK, Kemball-Cook G, Mosley KL, Mitrophanous K, Radcliffe P, Holder M, Brittan M, Georgiadis T, Al-Allaf F, Bigger B, Gregory L, Cook HT, Ali RR, Thrasher A, Tuddenham EGD, Themis M, Coutelle C (2004). Permanent phenotypic correction of Haemophilia B in immunocompetent mice by prenatal gene therapy.
    • Waddington SN, Nivsarkar MS, Mistry AR, Buckley SM, Kemball-Cook G, Mosley KL, Mitrophanous K, Radcliffe P, Holder MV, Brittan M, Georgiadis A, Al-Allaf F, Bigger BW, Gregory LG, Cook HT, Ali RR, Thrasher A, Tuddenham EG, Themis M, Coutelle C (2004). Permanent phenotypic correction of hemophilia B in immunocompetent mice by prenatal gene therapy.. Blood, 104(9), 2714 - 2721. doi:10.1182/blood-2004-02-0627
    • Ali RR (2004). Prospects for Gene Therapy. Novartis Foundation Symposium, 255, 165 - 172.
    • Dhomen N, Balaggan K, Bainbridge J, Ali R, Sowden J (2004). Lack of Chx10 causes embryonic retinal progenitors to persist in the adult retina.
    • Gaspar HB, Parsley K, Howe S, King D, Gilmour KC, Sinclair J, White H, Smith J, Barington T, Jacobsen MJ, Christensen HO, Brouns G, AlGhonaim A, Schmidt M, Von Kalle C, Ali RR, Kinnon C, Thrasher AJ (2004). Successful gene therapy of SCID-X1 using a pseudotyped gammaretroviral vector. The Lancet, 364(9452), 2181 - 2187. doi:10.1016/S0140-6736(04)17590-9
    • Schlichtenbrede FC, Tschernutter M, Bainbridge JB, Buch P, Georgiadis A, Smith AJ, Ali RR (2004). Gene therapy leads to long term improvement of retinal function in the heterozygous retinal degeneration slow mouse (rds(+/-)).
    • Joyce NC, Harris DL, Mc Alister JC, Ali RR, Larkin DF (2004). Effect of overexpressing the transcription factor E2F2 on cell cycle progression in rabbit corneal endothelial cells.. Investigative Ophthalmology and Visual Science, 45(5), 1340 - 1348.
    • Schlichtenbrede FC, Tschernutter M, Bainbridge JB, Buch P, Georgiadis A, Smith AJ, Ali RR (2004). Gene therapy leads to long term improvement of retinal function in the heterozygous retinal degeneration slow mouse (rds(+/-)). Investigative Ophthalmology and Visual Science, 45, 3706 - .
    • Bigger BW, Siapati KE, Mistry A, Waddington SN, Nivsarkar MS, Holder MV, Ridler C, Kemball-Cook G, Ali RR, Forbes S, Coutelle C, Alison M, Wright N, Thrasher AJ, Bonnet D, Themis M (2004). Induction of immune tolerance and partial phenotypic correction of haermophiliac mice by lentiviral gene transfer of human factor IX to haematopoietic stem cells. The Journal of Gene Medicine, 6(9), 38 - .
    • Dhomen NS, Balaggan KS, Bainbridge JW, Ali RR, Sowden JC (2004). Chx10 as a regulator of retinal stem cells.

    2003

    • Bainbridge JWB, Mistry A, Binley K, De Alwis M, Thrasher A, Naylor S, Ali RR (2003). Hypoxia-regulated transgene expression in experimental retinal and choroidal neovascularisation. Gene Therapy, 10(12), 1049 - 1054. doi:10.1038/sj.gt.3301945
    • Bainbridge JBL, Mistry A, Thrasher A, Ali RR (2003). Gene therapy for ocular angiogenesis. Clinical Science, 104(6), 561 - 575.
    • Smith AJ, Schlichtenbrede FC, Tschernutter M, Bainbridge JW, Thrasher AJ, Ali RR (2003). AAV-Mediated gene transfer slows photoreceptor loss in the RCS rat model of retinitis pigmentosa. Molecular Therapy, 8(2), 188 - 195.
    • Bainbridge JW, Jia H, Bagherzadeh A, Selwood D, Ali RR, Zachary I (2003). A peptide encoded by exon 6 of VEGF (EG3306) inhibits VEGF-induced angiogenesis in vitro and ischaemic retinal neovascularisation in vivo. Biochemical and Biophysical Research Communications, 302(4), 793 - 799.
    • Ali RR, Sowden JC (2003). Therapy may yet stem from cells in the retina. Br J Ophthalmol, 87(9), 1058 - 1059.
    • Bainbridge JWB, Mistry A, Schlichtenbrede FC, Smith A, Broderick C, De Alwis M, Georgiadis A, Taylor PM, Squires M, Sethi C, Charteris D, Thrasher AJ, Sargan D, Ali RR (2003). Stable rAAV-mediated transduction of rod and cone photoreceptors in the canine retina. Gene Therapy, 10(16), 1336 - 1344. doi:10.1038/sj.gt.3301990
    • Hughes EH, Schlichtenbrede FC, Murphy CC, Sarra GM, Luthert PJ, Ali RR, Dick AD (2003). Generation of activated sialoadhesin-positive microglia during retinal degeneration. Investigative Ophthalmology and Visual Science, 44(5), 2229 - 2234.
    • Ali RR (2003). Gene therapy for inherited retinal degeneration.
    • Cordeiro MF, Mead A, Ali RR, Alexander RA, Murray S, Chen C, York-Defalco C, Dean NM, Schultz GS, Khaw PT (2003). Novel antisense oligonucleotides targeting TGF-beta inhibit in vivo scarring and improve surgical outcome. Gene Therapy, 10(1), 59 - 71. doi:10.1038/sj.gt.3301865
    • Hughes EH, Schlichtebrede FC, Murphy CC, Ali RR, Dick AD (2003). Minocycline suppresses photoreceptor apoptosis in the rds mouse through a mechanism unrelated to microglial inhibition.
    • Schlichtenbrede FC, da Cruz L, Stephens C, Smith AJ, Georgiadis A, Thrasher AJ, Bainbridge JW, Seeliger MW, Ali RR (2003). Long-term evaluation of retinal function in Prph2Rd2/Rd2 mice following AAV-mediated gene replacement therapy. The Journal of Gene Medicine, 5(9), 757 - 764.
    • Schlichtenbrede FC, MacNeil A, Bainbridge JW, Tschernutter M, Thrasher AJ, Smith AJ, Ali RR (2003). Intraocular gene delivery of ciliary neurotrophic factor results in significant loss of retinal function in normal mice and in the Prph2Rd2/Rd2 model of retinal degeneration. Gene Therapy, 10(6), 523 - 527.

    2002

    • Schlichtenbrede FC, Sarra GM, Ali RR, Wiedemann P, Reichel MB (2002). Progress in the somatic gene therapy of retinal dystrophies in animal models. OPHTHALMOLOGE, 99(4), 259 - 265. doi:10.1007/s00347-002-0633-x
    • Mistry AR, De Alwis M, Feudner E, Ali RR, Thrasher AJ (2002). High-titer stocks of adeno-associated virus from replicating amplicons and herpes vectors. Methods in Molecular Medicine, 69, 445 - 460.
    • Binley K, Askham Z, Iqball S, Spearman H, Martin L, De Alwis M, Thrasher AJ, Ali RR, Maxwell PH, Kingsman S, Naylor S (2002). Long-term reversal of chronic anemia using a hypoxia-regulated erythropoietin gene therapy. BLOOD, 100(7), 2406 - 2413.
    • Sarra GM, Stephens C, Schlichtenbrede FC, Bainbridge JW, Thrasher AJ, Luthert PJ, Ali RR (2002). Kinetics of transgene expression in mouse retina following sub-retinal injection of recombinant adeno-associated virus. Vision Research, 42(4), 541 - 549.
    • Bainbridge JWB, Mistry A, De Alwis M, Paleolog E, Baker A, Thrasher AJ, Ali RR (2002). Inhibition of retinal neovascularisation by gene transfer of soluble VEGF receptor sFlt-1. GENE THER, 9(5), 320 - 326. doi:10.1038/sj/gt/3301680
    • Bainbridge JWB, Mistry A, De Alwis M, Paleolog E, Baker A, Thrasher AJ, Ali RR (2002). Inhibition of retinal neovascularisation by gene transfer of soluble VEGF receptor sFlt-1. Gene Therapy, 9(5), 320 - 326. doi:10.1038/sj.gt.3301680
    • Schlichtenbrede FC, Sarra GM, Ali RR, Wiedemann P, Reichel MB (2002). [Progress in somatic gene therapy of retinal degeneration in the animal model]. Der Ophthalmologe, 99(4), 259 - 265.

    2001

    • (2001). Molecular Genetics and Prospects for Therapy of the Inherited Retinal Dystrophies. Current Opinion in Genetics and Development, 11(3), 307 - 316.
    • Sarra GM, Stephens C, De Alwis M, Bainbridge JW, Smith AJ, Thrasher AJ, Ali RR (2001). Gene replacement therapy in the retinal degeneration slow (rds) mouse: the effect on retinal degeneration following partial transduction of the retina. Human Molecular Genetics, 10(21), 2353 - 2361.
    • Ali RR, Sarra GM, Stephens CM, de Alwis M, Bainbridge JWB, Thrasher AJ (2001). Gene therapy for inherited retinal degeneration. J MED GENET, 38, S26 - S26.
    • (2001). In vivo gene tranfer to the mouse eye using an HIV-based lentiviral vector; efficient long term transduction of corneal endothelium and retinal pigment epithelium. Gene Therapy, 8(21), 1665 - 1668. doi:10.1038/sj.gt.3301574
    • (2001). Genetic incorporation of the SIGYPLP peptide into adenoassociated virus-2 capsids directs efficient and selective gene transfer to the human vascular endothelial cells. Molecular Therapy, 4, 174 - 181.
    • Sarra G, Stephens C, De Alwis M, Bainbridge J, Munro P, Thrasher AJ, Ali RR (2001). Long term restoration of photoreceptor outer segments in the rds mouse after gene replacement therapy. Investigative Ophthalmology and Visual Science, 42(4), 1878 - .
    • Feudner E, De Alwis M, Thrasher AJ, Ali RR, Fauser S (2001). Optimization of recombinant adeno-associated virus production using an herpes simplex virus amplicon system. Journal of Virological Methods, 96(2), 97 - 105.
    • Reichel MB, Bainbridge J, Baker D, Thrasher AJ, Bhattacharya SS, Ali RR (2001). An immune response after intraocular administration of an adenoviral vector containing a beta galactosidase reporter gene slows retinal degeneration in the rd mouse. British Journal of Ophthalmology, 85(3), 341 - 344.
    • Nicklin SA, Buening H, Dishart KL, De Alwis M, Girod A, Hacker U, Thrasher AJ, Ali RR, Hallek M, Baker AH (2001). Efficient and selective AAV2-mediated gene transfer directed to human vascular endothelial cells. Molecular Therapy, 4(3), 174 - 181.

    2000

    • (2000). Restoration of photoreceptor ultrastructure and function in retinal degeneration slow mice by gene therapy. Nature Genetics, 25(3), 306 - 310.
    • Reichel MB, Paul D, Holborn M, Schlichtenbrede FC, Ali RR, Wiedemann P (2000). Caspases are overexpressed during photoreceptor cell apoptosis in the RD and RDS mouse.. INVEST OPHTH VIS SCI, 41(4), S403 - S403.

    1999

    • Reichel MB, Hudde T, Ali RR, Wiedemann P (1999). [Gene transfer in ophthalmology]. Der Ophthalmologe, 96(9), 570 - 577.
    • Bainbridge J, Stephens C, Zhang Z, Reichel MB, Thrasher AJ, Ali RR (1999). Efficient transduction of photoreceptor cells using high titre rAAV produced from replicating amplicons and gH- herpes vectors. Investigative Ophthalmology and Visual Science, 40, 44747 - .
    • Cordeiro MF, Schultz GS, Ali RR, Bhattacharya SS, Khaw PT (1999). Molecular therapy in ocular wound healing. British Journal of Ophthalmology, 83(11), 1219 - 1224.
    • (1999). High frequency of persistent hyperplastic primary vitreous and cataracts in p53-deficient mice. Cell Death and Differentiation, 5(2), 156 - 162.

    1998

    • Ali RR, Reichel MB, De Alwis M, Kanuga N, Kinnon C, Levinsky RJ, Hunt DM, Bhattacharya SS, Thrasher AJ (1998). Adeno-associated virus gene transfer to mouse retina.. Hum Gene Ther, 9(1), 81 - 86. doi:10.1089/hum.1998.9.1-81
    • Ali RR, Reichel MB, Kanuga N, Munro PM, Alexander RA, Clarke AR, Luthert PJ, Bhattacharya SS, Hunt DM (1998). Absence of p53 delays apoptotic photoreceptor cell death in the rds mouse.. Curr Eye Res, 17(9), 917 - 923.
    • Adams MJ, Reichel MB, King IA, Marsden MD, Greenwood MD, Thirwell H, Arnemann J, Buxton RS, Ali RR (1998). Characterization of the regulatory regions in the human desmoglein genes encoding the pemphigus foliaceous and pemphigus vulgaris antigens. BIOCHEM J, 329, 165 - 174.
    • Croxford JL, O'Neill JK, Ali RR, Browne K, Byrnes AP, Dallman MJ, Wood MJA, Feldmann M, Baker D (1998). Local gene therapy with CTLA4-immunoglobulin fusion protein in experimental allergic encephalomyelitis. European Journal of Immunology, 28(12), 3904 - 3916.
    • Ali RR, Reichel MB, de Alwis M, Kanuga N, Kinnon C, Levinsky RJ, Hunt DM, Bhattacharya SS, Thrasher AJ (1998). Adeno-associated virus gene transfer to mouse retina. Human Gene Therapy. Human Gene Therapy, 9, 81 - 86.
    • Ali RR, Reichel MB, Kanuga N, Clarke AR, Luthert PJ, Bhattacharya SS, Hunt DM (1998). Gene therapy for inherited retinal degeneration.. British Journal of Ophthalmology, 81, 795 - 801.
    • Ali RR, Reichel MB, Baker D, Byrnes AP, Kanuga N, Hunt DM, Bhattacharya SS (1998). Co-injection of adenovirus expressing CTLA4-Ig prolongs adenovirally-mediated LacZ reporter gene expression in the mouse retina.. Gene Therapy, 5(11), 1561 - 1565.
    • Hart SL, Arancibia-Cárcamo CV, Wolfert MA, Mailhos C, O'Reilly NJ, Ali RR, Coutelle C, George AJ, Harbottle RP, Knight AM, Larkin DF, Levinsky RJ, Seymour LW, Thrasher AJ, Kinnon C (1998). Lipid-mediated enhancement of transfection by a nonviral integrin-targeting vector.. Hum Gene Ther, 9(4), 575 - 585. doi:10.1089/hum.1998.9.4-575
    • Reichel MB, Ali RR, Hunt DM, Bhattacharya SS (1998). Gene therapy for retinal degeneration.. Ophthalmic Research, 29, 261 - 268.
    • Reichel MB, Ali RR, D Esposito F, Clarke AR, Luthert PJ, Bhattacharya SS, Hunt DM (1998). High frequency of hyperplastic primary vitreous in p53-deficient mice.. Cell Death and Differentiation, 5, 156 - 162.
    • Reichel MB, Ali RR, Thrasher AJ, Hunt DM, Bhattacharya SS, Baker D (1998). Immune responses limit adenovirally-mediated gene expression in the adult mouse eye.. Gene Therapy, 5(8), 1038 - 1046.

    1997

    • Reichel MB, Ali RR, Baker D, Byrnes AP, DEsposito F, Kanuga N, Hunt DM, Bhattacharya SS (1997). Co-injection of adenovirus expressing CTLA4-Ig prolongs adenovirally-mediated lacZ reporter gene expression in the mouse retina.. INVEST OPHTH VIS SCI, 38(4), 1198 - 1198.
    • Ali RR, Reichel MB, Thrasher AJ, Kanuga N, Hunt DM, Bhattacharya SS (1997). Gene delivery to the RDS mouse. INVEST OPHTH VIS SCI, 38(4), 1194 - 1194.

    1996

    • Ali RR, Reichel MB, Thrasher AP, Kanuga N, Hunt DM, Bhattacharya SS (1996). In vivo gene delivery to the mouse retina. INVEST OPHTH VIS SCI, 37(3), 41 - 41.
    • Ali RR, Reichel MB, Thrasher AJ, Levinsky RJ, Kinnon C, Kanuga N, Hunt DM, Bhattacharya SS (1996). Gene transfer into the mouse retina mediated by an adeno-associated viral vector.. Hum Mol Genet, 5(5), 591 - 594.

    • Barber AC, Hippert C, West EL, Duran Y, Bainbridge JWB, Warre-Cornish K, Luhmann UFO, Lakowski J, Sowden JC, Ali RR, Pearson RA (). Repair of the degenerate retina by photoreceptor transplantation. Proceedings of the National Academy of Sciences of USA, , - .