Publications

Select publications in each of our main areas of research

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

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

Gene and cell therapies for age-related macular degeneration (AMD) and diabetic eye disease

Gene therapy to control the damaging growth of blood vessels

Inhibition of retinal neovascularisation by gene transfer of soluble VEGF receptor sFlt-1. JWB Bainbridge, A Mistry, M De Alwis, E Paleolog, A Baker, AJ Thrasher and RR Ali Gene Ther. 2002; 9 (5): 320-326

EIAV vector-mediated delivery of endostatin or angiostatin inhibits angiogenesis and vascular hyperpermeability in experimental CNV. KS Balaggan, K Binley, M Esapa, Re MacLaren, S Iqball, Y Duran, RA Pearson, O Kan, SE Barker, AJ Smith, JWB Bainbridge, S Naylor and RR Ali Gene Ther. 2006; 13 (15):1153-1165

Models of disease

Differential modulation of retinal degeneration by Ccl2 and Cx3cr1 chemokine signalling. UF Luhmann, CA Lange, S Robbie, PM Munro, JA Cowing, HE Armer, V Luong, LS Carvalho, RE MacLaren, FW Fitzke, JW Bainbridge and RR Ali PLoS One 2012; 7 (4): e35551

The Drusen-like phenotype in aging Ccl2 knockout mice is caused by an accelerated accumulation of swollen autofluorescent subretinal macrophages. UF Luhmann, S Robbie, PM Munro, SE Barker, Y Duran V Luong, FW Fitzke, J Bainbridge, RR Ali & R MacLaren. Invest Ophthalmol Vis Sci. 2009; 50 (12): 5934-43

Von Hippel-Lindau protein in the RPE is essential for the normal ocular growth and vascular development. CAK Lange, UFO Luhmann, FM Moway, EL West, H Sayed, AJ Smith, JC Sodewn, PH Maxwell, RR Ali and JW Bainbridge Development 2012; 139 (13): 2340-50

HIF-1alpha and HIF-2alpha are differentially activated in distinct cell populations in retinal ischaemia. FM Mowat, UF Luhmann, AJ Smith, C Lange, Y Duran, S Harten, D Shukla, PH Maxwell, RR Ali and JW Bainbridge. PLoS One 2010; 5 (6): e11103

Clinical disease mechanisms

Intraocular oxygen distribution in advanced poliferative diabetic retinopathy.
CA Lange, P Stavrakas, UF Luhmann, DJ de Silva, RR Ali, ZJ Gregor & JW Bainbridge Am J Ophthalmol. 2011;152 (3): 406-412

Gene therapy for uveitis and corneal disease

Gene therapy for non-infectious uveitis. Chu CJ, Barker SE, Dick AD and Ali RR. Ocular Immunol and Inflam. 2012 (in press)

Lentiviral-vector mediated expression of murine IL-1 receptor antagonist or IL-10 reduces the severity of endotoxin-induced uveitis. P Trittibach, SE Barker, CA Broderick, M Natkunaraja, Y Duran, SJ Robbie, JWB Bainbridge, G-M Sarra, AD Dick and RR Ali Gene Ther. 2008; 15 (22): 1478-88

Local administration of an adeno-associated viral vector expressing IL-10 reduces monocyte infiltration and subsequent photoreceptor damage during experimental autoimmune uveitis. CA Broderick, AJ Smith, KS Balaggan, A Georgiadis, PK Buch, PC Trittibach, SE Barker, G Sarra, AJ Thrasher, AD Dick and RR Ali. Mol. Ther. 2005; 12 (2): 369-373

Full publication list

2013

Assessment and in vivo scoring of murine experimental autoimmune uveoretinitis using optical coherence tomography Chu CJ, Herrmann P, Carvalho LS, Liyanage SE, Bainbridge JW, Ali RR, Dick AD, Luhmann UF. PLoS One. 2013 May 14;8(5):e63002

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) Luhmann UF, Carvalho LS, Robbie SJ, Bainbridge JW, Ali RR. Exp Eye Res. 2013 Jun;111:136

Repair of the degenerate retina by photoreceptor transplantation. Barber AC, Hippert C, Duran Y, West EL, Bainbridge JWB, Warre-Cornish K, Luhmann UFO, Sowden JC, Ali RR, Pearson RA. Proc. Natl. Acad. Sci. 2013 Jan 2;110(1):354-9.

Subconjunctival bevacizumab induces regression of corneal neovascularisation: a pilot randomised placebo-controlled double-masked trial. Petsoglou C, Balaggan KS, Dart JK, Bunce C, Xing W, Ali RR, Tuft SJ. Br J Ophthalmol. 2013 Jan;97(1):28-32.

2012

CCl2, Cx3cr1 and Ccl2/Cx3cr1 chemokine deficiencies are not sufficient to cause age-related retinal degeneration. UFO Luhmann, LS Carvalho, SJ Robbie, JA Cowing, Y Duran, PM Munro, JW Bainbridge, RR Ali Exp. Eye Res. 2012 Dec 8;107C:80-87.

Gene therapy for noninfectious uveitis. Chu CJ, Barker SE, Dick AD, Ali RR. Ocul Immunol Inflamm. 2012 Dec;20(6):394-405.

Self-organising neuroepithelium from human pluripotent stem cells facilitates derivation of photoreceptors. C Boucherie, S Mukherjee, E Henckaerts, AJ Thrasher, JC Sowden and RR Ali. Stem Cells 2012 Nov 6.

Successful gene therapy in older RPE65-deficient dogs following subretinal injection of an adeno-associated vector expressing RPE65. MJ Annear, JT Bartoe, PG Curran, AJ Smith, JW Bainbridge, RR Ali and SM Petersen-Jones. Hum Gene Ther (under revision)

RPE65 gene therapy slows cone loss in Rpe65-deficient dogs. FM Mowat, AR Breuwer, JT Bartoe, MJ Annear, Z Zhang, AJ Smith, JW Bainbridge, SM Petersen-Jones and RR Ali Gene Ther. 2012 [Epub ahead of print]

Defining the integration capacity of embryonic stem cell-derived photoreceptor precursors. EL West, A Gonzalez-Cordero, C Hippert, F Osakada, RA Pearson, JC Sodwen, M Takahashi and RR Ali Stem Cells 2012; 30 (7): 1424-35

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.

Differential modulation of retinal degeneration by Ccl2 and Cx3cr1 chemokine signalling. UF Luhmann, CA Lange, S Robbie, PM Munro, JA Cowing, HE Armer, V Luong, LS Carvalho, RE MacLaren, FW Fitzke, JW Bainbridge and RR Ali PLoS One 2012; 7 (4): e35551

Gene therapy for retinal dystrophies: twenty years in the making. RR Ali Hum Gene Ther. 2012; 23 (4): 337-9

Leber congenital amaurosis associated with AIPL1: challenges in ascribing disease causation, clinical findings, and implications for gene therapy. MH Tan, DS Mackay, J Cowing, HV Tran, AJ Smith, GA Wright, A Dev-Borman, RH Henderson, P Moradi, I Russell-Eggitt, RE MacLaren, AG Robson, ME Cheetham, DA Thompson, AR Webster, M Michaelides, RR Ali and AT Moore. PLoS One 2012; 7 (3): e32330

Von Hippel-Lindau protein in the RPE is essential for the normal ocular growth and vascular development. CAK Lange, UFO Luhmann, FM Moway, EL West, H Sayed, AJ Smith, JC Sodewn, PH Maxwell, RR Ali and JW Bainbridge Development 2012; 139 (13): 2340-50

Endogenous Erythropoietin Protects Neuroretinal Function in Ischemic Retinopathy.
FM Mowat, F Gonzalez, UF Luhmann, C Lange, Y Duran, AJ Smith, PH Maxwell, RR Ali, JW Bainbridge. Am J Pathol. 2012 [Epub ahead of print]

Manipulation of the recipient retinal environment by ectopic expression of neurotrophic growth factors can improve transplanted photoreceptor integration and survival.
EL West, RA Pearson, Y Duran, A Gonzalez-Cordero, RE Maclaren, AJ Smith, JC Sowden, RR Ali. Cell Transplant.2012 [Epub ahead of print]

Ocular gene therapy: introduction to the special issue.

RR Ali. Gene Ther. 2012; 19 (2): 119-20

Gene augmentation trials using the rpe65-deficient dog: contributions towards development and refinement of human clinical trials.

SM Petersen-Jones, MJ Annear, JT Bartoe, FM Mowat, SE Barker, AJ Smith, JW Bainbridge and RR Ali Adv Exp Med Biol. 2012; 723: 177-82

Local Vs. systemic mononuclear phagocytes in age-related macular degeneration and their regulation by CCL2-CCR2 and CX3CL1-CX3CR1 chemokine signaling.
UF Luhmann and RR Ali Adv Exp Med Biol. 2012; 723: 17-22

Absence of ocular malignant transformation after sub-retinal delivery of rAAV2/2 or integrating lentiviral vectors in p53-deficient mice.
KS Balaggan, Y Duran, A Georgiadis, C Thaung, SE Barker, PK Buch, A MacNeil, S Robbie, JW Bainbridge, AJ Smith and RR Ali Gene Ther.2012; 19 (2): 182-8

Educational paper: Retinal dystrophies and gene therapy. V Sundaram, AT Moore, RR Ali and JW Bainbridge Eur J Pediatr. 2012; 171 (5): 757-65

Isolation and culture of adult ciliary epithelial cells, previously identified as retinal stem cells, and retinal progenitor cells. S Gualdoni, M Baron, J Lakowski, S Decembrini, RA Pearson, RR Ali and JC Sowden. Curr Protoc Stem Cell Biol. 2011 Dec; Chapter 1: Unit 1H.4.

Ocular gene delivery using lentiviral vectors. KS Balaggan and RR Ali Gene Ther. 2012; 19 (2): 145-53

Gene supplementation therapy for recessive forms of inherited retinal dystrophies.
AJ Smith, JW Bainbridge and RR Ali Gene Ther. 2012; 19 (2): 154-61

Long-term preservation of cones and improvement in visual function following gene therapy in a mouse model of Leber congenital amaurosis (LCA) caused by GC1 deficiency. M Mihelec, RA Pearson, SJ Robbie, PK Buch, WB Bainbridge, AJ Smith and RR Ali Human Gene Therapy  2011; 22 (10): 1179-90

Experimental gene transfer to the corneal endothelium.
D Kampik, RR Ali & DF Larkin Exp Eye Res. 2012; 95 (1): 54-9

2011


Effective transplantation of photoreceptor precursor cells selected via cell surface antigen expression.
J Lakowski, YT Han, RA Pearson, A Gonzalez-Cordero, EL West, S Gualdoni, AC Barber, M Hubank, RR Ali and JC Sowden Stem Cells. 2011; 29 (9): 1391-404

Characterisation of a C1qtnf5 Ser163Arg knock-in mouse model of late-onset retinal macular degeneration. Shu X, Luhmann UF, Aleman TS, Barker SE, Lennon A, Tulloch B, Chen M, Xu H, Jacobson SG, Ali R, Wright AF. PLoS One. 2011; 6 (11):e27433. 

Induced pluripotent stem cell technology for generating photoreceptors.
C Boucherie, JC Sowden & RR Ali Regen Med. 2011; 6 (4): 469-79

Intraocular oxygen distribution in advanced poliferative diabetic retinopathy.
CA Lange, P Stavrakas, UF Luhmann, DJ de Silva, RR Ali, ZJ Gregor & JW Bainbridge Am J Ophthalmol. 2011;152 (3): 406-412

Gene therapy in the second eye of RPE65 deficient dogs improves retinal function.

MJ Annear, JT Bartoe, SE Barker, AJ Smith, PG Curran, JW Bainbridge, RR Ali and SM Petersen-Jones.Gene Ther. 2011; 18 (1): 53-61 

Comparative analysis of the retinal potential of embryonic stem cells and amniotic fluid-derived stem cells.
S Decembrini, M Cananzi, S Gualdoni, A Battersby, N Allen, RA Pearson, RR Ali, P De Coppi and JC Sowden Stem Cells Dev. 2011; 20 (5): 851-63

Isolation and culture of adult ciliary epithelial cells, previously identified as retinal stem cells, and retinal progenitor cells.
S Gualdoni, M Baron, J Lakowski, S Decembrini, RA Pearson, RR Ali, JC Sowden. Curr Protoc Stem Cell Biol. 2011

Long-term preservation of cones and improvement in visual function following gene therapy in a mouse model of Leber congenital amaurosis (LCA) caused by GC1 deficiency.

M Mihelec, RA Pearson, SJ Robbie, PK Buch, WB Bainbridge, AJ Smith and RR Ali Human Gene Therapy  2011 [Epub ahead of print]

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

Regenerative medicine: DIY eye.

RR Ali and JC Sowden. Nature 2011; 472 (7341): 42-3 

Dominant cone-rod dystrophy: a mouse model generated by gene targeting of the GCAP1/Guca1a gene.

PK Buch, M Mihelec, P Cottrill, SE Wilkie, RA Pearson, Y Duran, EL West, M Michaelides, RR Ali and DM Hunt.  PLos One 2011; 6 (3): e18089

Lentiviral vector integration profiles differ in rodent postmitotic tissues.
CC Bartholomae, A Arens, KS Balaggan, RJ Yáñez-Muñoz, E Montini, SJ Howe, A Paruzynski, B Korn, JU Appelt, A Macneil, D Cesana, U Abel, H Glimm, L Naldini, RR Ali, AJ Thrasher, C von Kalle and M Schmidt Mol Ther. 2011; 19 (4): 703-10

2010

The tight junction associated signalling proteins ZO-1 and ZONAB regulate retinal pigment epithelium homeostasis in mice.
A Georgiadis, M Tschernutter, JW Bainbridge, KS Balaggan, F Mowat, EL West, PM Munro, AJ Thrasher, K Matter, MS Balda and Ali RRPLoS One 2010; 5 (12): e15730 

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 

Airbag injury and bilateral globe rupture.
T Salam, P Stavrakas, L Wickham and J Bainbridge. Am J Emerg Med. 2010; 28 (8): 982 e5-6 

Long Term Survival of Photoreceptors Transplanted into the Adult Murine Neural Retina Requires Immune Modulation.
EL West, RA Pearson, SE Barker, UF Luhmann, RE MacLaren, AC Barber, Y Duran, AJ Smith, JC Sowden and RR Ali. Stem Cells. 2010; 28 (11): 1997-2007

GFAP-driven GFP expression in activated mouse Müller glial cells aligning retinal blood vessels following intravitreal injection of AAV2/6 vectors.

WM Aartsen, KW van Cleef, LP Pellissier, RM Hoek, RM Vos, B Blits, EM Ehlert, KS Balaggan, RR Ali, J Verhaagen and J Wijnholds PLoS One. 2010; 5 (8): e12387 

Gene therapy in the second eye of RPE65-deficient dogs improves retinal function.

MJ Annear, JT Bartoe, SE Barker, AJ Smith, PG Curran, JW Bainbridge, RR Ali and SM Petersen-Jones. Gene Ther. 2010 [Epub ahead of print]

HIF-1alpha and HIF-2alpha are differentially activated in distinct cell populations in retinal ischaemia. FM Mowat, UF Luhmann, AJ Smith, C Lange, Y Duran, S Harten, D Shukla, PH Maxwell, RR Ali and JW Bainbridge. PLoS One 2010; 5 (6): e11103 

Adult ciliary epithelial cells, previously identified as retinal stem cells with potential for retinal repair, fail to differentiate into new rod photoreceptors
.
S Gualdoni, M Baron, J Lakowski, S Decembrini, AJ Smith, RA Pearson, RR Ali and JC Sowden Stem Cells 2010; 28 (6): 1048-59

Delivery of anti-angeogenic molecular therapies for retinal disease.

OA Anderson, JW Bainbridge, DT Shima Drug Discov Today 2010; 15 (7-8): 272-82 

Targeted disruption of outer limiting membrane junctional proteins (Crb1 and ZO-1) increases integration of transplanted photoreceptor precursors into the adult wildtype and degenerating retina.
R Pearson, A Barber, E West, RE MacLaren, Y Duran, JW Bainbridge, J Sowden and RR Ali Cell Transplant. 2010; 19 (4): 487-503 

Gene therapy with a promotor targeting both rods and cones rescues retinal degeneration caused by AIPL1 mutations
.
X Sun, B Pawlyk, X Xu, X Liu, OV Bulgakov, M Adamian, MA Sandberg, SC Khani, MH Tan, AJ Smith, RR Ali and T Li. Gene Ther. 2010; 17 (1): 117-31

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

A pilot randomised controlled trial comparing the post-operative pain experience following vitrectomy with a 20-gauge and the 25-gauge transconjuntival system.
L Wickham, C Bunce, AS Kwan, J Bainbridge and GW Aylward Br J Ophthalmol. 2010; 94 (1): 36-40 

2009


Comprehensive genomic access to vector integration in clinical gene therapy.
R Gabriel, R Eckenberg, A Paruzynski, CC Bartholomae, A Nowrouzi, A Arens, SJ Howe, A Recchia, C Cattoglio, W Wang, K Faber, K Schwarzwaelder, R Kirsten, A Deichmann, CR Ball, KS Balaggan, RJ Yáñez-Muñoz, RR Ali, HB Gaspar, L Biasco, A Aiuti, D Cesana, E Montini, L Naldini, O Cohen-Haguenauer, F Mavilio, AJ Thrasher, H Glimm, C von Kalle, W Saurin and M Schmidt Nat Med. 2009; 15 (12): 1431-6

Cell Transplantation strategies for retinal repair.
EL West, RA Pearson, RE MacLaren, JC Sowden and RR Ali Prog Brain Res. 2009; 175: 3-21

The Drusen-like phenotype in aging Ccl2 knockout mice is caused by an accelerated accumulation of swollen autofluorescent subretinal macrophages.
UF Luhmann, S Robbie, PM Munro, SE Barker, Y Duran V Luong, FW Fitzke, J Bainbridge, RR Ali & R MacLaren. Invest Ophthalmol Vis Sci. 2009; 50 (12): 5934-43

Prospects for gene therapy of inherited retinal disease
.
JWB Bainbridge Eye 2009; 23 (10): 1898-903

Subretinal delivery of adeno-associated virus serotype 2 results in minimal immune responses that allow repeat vector administration in immunocompetent mice.
SE Barker, CA Broderick, SJ Robbie, Y Duran, M Natkunarajah, P Buch, KS Balaggan, RE MacLaren, JW Bainbridge, AJ Smith and RR Ali J Gene Med. 2009; 11 (6): 486-97 

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

Complement factor h is critical in the maintenance of retinal perfusion.
P Lundh con Leithner, JH Kam, J Bainbridge, I Catchpole, G Gough, P Coffey and G Jeffrey Am J Pathol 2009; 175 (1): 412-21 

Prospects for retinal gene therapy.
AJ Smith, JW Bainbridge and RR Ali Trends Genet . 2009; 25 (4): 156-65

2008


Lentiviral-vector mediated expression of murine IL-1 receptor antagonist or IL-10 reduces the severity of endotoxin-induced uveitis.
P Trittibach, SE Barker, CA Broderick, M Natkunaraja, Y Duran, SJ Robbie, JWB Bainbridge, G-M Sarra, AD Dick and RR Ali Gene Ther. 2008; 15 (22): 1478-88 

Success in sight: The eyes have it! Ocular gene therapy trials for LCA look promising.
JW Bainbridge and RR Ali Gene Ther. 2008; 15 (17): 1191-2 

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

Isolation and characterisation of neural progenitor cells from the adult Chx10(orJ/orJ) central neural retina.
I Kokkinopoulos, RA Pearson, A MacNeil, NS Dohman, RE MacLaren, RR Ali and JC SowdenMol. Cell Neurosci. 2008; 38 (3): 359-73 

AAV-mediated gene therapy for retinal disorders: from mouse to man.
PK Buch, JW Bainbridge and RR Ali. Gene Ther. 2008; 15 (11): 849-57 

Keeping an eye on clinical trials in 2008.
JW Bainbrisge and RR Ali. Gene Ther. 2008; 15 (9): 633-4 

Pharmacological disruption of the outer limiting membrance leads to increased retinal integration of transplanted photoreceptor precursors.
EL West, RA Pearson, M Tschernutter, JC Sowden, RE MacLaren and RR Ali Exp. Eye Res. 2008; 86 (4): 601-11 

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 

Topographical characterization of cone photoreceptors and the area centralis of the canine retina.
FM Mowat, SM Petersen-Jones, H Williamson, DL Williams, PJ Luthert, RR Ali and JW Bainbridge Mol Vis. 2008; 14: 2518-27 

Control of cell proliferation by neurotransmitters in the developing vertebrate retina. RA Martins and RA Pearson Brain Res. 2008; 1192: 37-60

2007


Gene transfer of an engineered zinc finger protein enhances the anti-angiogenic defense system.
K Yokoi, HS Zhang, S Kachi, KS Balaggan, Q Yu, D Guschin, M Kunis, R Surosky, LM Africa, JW Bainbridge, SK Spratt, PD Gregory, RR Ali and PA Campochiaro Mol Ther. 2007; 15 (11): 1917-23 

Progress and prospects: gene therapy clinical trials (part 1).
BL Alexander, RR Ali, EW Alton, JW Bainbridge, S Braun, SH Cheng, TR Flotte, HB Gaspar, U Grez, M Griesenbach, MG Kaplitt, MG Ott, R Segar, M Simons, AJ Thrasher and S Yla-Herttuala Gene Ther. 2007; 14 (20): 1439-47 

The macrophage is the key to choroidal neovascularization in age-related macular degeneration.
JWB Bainbridge Exp. Rev. Ophthalmol. 2007; 2 (6): 981-86 

Gene therapy clinical trials for inherited eye disease.
JWB Bainbridge Exp. Rev. Ophthalmol. 2007; 2 (4): 517-19 

Neuroprotective gene therapy for the treatment of inherited retinal degeneration.
P Buch, RE MacLaren and RR Ali Curr Gen Ther. 2007; 7 (6): 434-45 

Stem cell therapy and the retina.
RE MacLaren and RA Pearson Eye. 2007; 21 (10):1352-9 

Comparative analysis of progenitor cells isolated from the iris, pars plana and ciliary body of the adult porcine eye.
A MacNeil, RA Pearson, RE MacLaren, AJ Smith, JC Sowden and RR Ali Stem Cells. 2007; 25 (10): 2430-8 

Biometry and formula accuracy with intraocular lenses used for cataract surgery in extreme hyperopia.
RE MacLaren, M Natkunarajah, Y Riaz, RR Bourne, M Restorie and BD Allan Am J Ophthalmol. 2007;143 (6): 920-31

AAV-mediated expression targeting of rod and cone photoreceptors with a human rhodopsin kinase promoter.
SC Khani, BS Pawlyk, OV Bulgakov, E Kasperek, JE Young, M Adamian, X Sun, AJ Smith, RR Ali and T Li Invest Ophthalmol Vis Sci. 2007; 48 (9): 3954-61 

Autologous transplantation of the retinal pigment epithelium and choroid in the treatment of neovascular age-related macular degeneration.
RE MacLaren, GS Uppal, KS Balaggan, AD Tufail, PM Munro, AB Milliken, RR Ali, GS Rubin, GW Aylward and L Da Cruz Ophthalmol 2007; 114 (3): 561-70

2006


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

Uveitic glaucoma and Rosai-Dorfman disease (sinus histiocytosis).
RE MacLaren, KS Hundal, P Trittibach and PA Bloom Ocul Immunol Inflamm. 2006; 14 (5): 305-7

CNTF gene transfer protects ganglion cells in rat retinae undergoing focal injury and branch vessel occlusion.
RE Maclaren, PK Buch, AJ Smith, KS Balaggan, A Macneil, JS Taylor, NN Osborne and RR AliExp. Eye Res. 2006; 83 (5): 1118-27

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

Gene Therapy progress and prospects: the eye.
JWB Bainbridge, MH Tan and RR Ali Gene Ther. 2006; 13 (16): 1191-1197

Inhibition of ocular Neovascularisation by Hedgehog Blockade
.
EM Surace, KS Balaggan, A Tessitore, C Mussolino, G Cotugno, C Bonetti, A Vitale, RR Ali and A Auricchio. Mol. Ther. 2006; 13 (3): 573-579

Regulation of PCNA and cyclin D1 expression and epithelial morphogenesis by the ZO-1-regulated transcript factor ZONAB/DbpA.
T Sourisseau, A Georgiadis, A Tsapara, RR Ali, R Pestell, K Matter and MS Balda Mol. Cell Biol. 2006; 26 (6): 2387-2398

Clinical characterisation of a family with retinal dystrophy caused by mutation of the Mertk gene.
M Tschernutter, SA Jenkins, N Waseem, Z Saihan, GE Holder, AC Bird, SS Bhattacharya, RR Ali and AR Webster Br. J. Ophthalmol. 2006; 90 (6):718-723

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

EIAV vector-mediated delivery of endostatin or angiostatin inhibits angiogenesis and vascular hyperpermeability in experimental CNV.
KS Balaggan, K Binley, M Esapa, Re MacLaren, S Iqball, Y Duran, RA Pearson, O Kan, SE Barker, AJ Smith, JWB Bainbridge, S Naylor and RR Ali Gene Ther. 2006; 13 (15):1153-1165

Permanent partial phenotypic correction and tolerance in a mouse model of hemophilia B by stem cell gene delivery of human factor IX.
BW Bigger, EK Siapati, AR Mistry, SN Waddington, MS Nivsarkar, L Jacobs, R Perrett, MV Holder, C Ridler, G Kemball-Cook, RR Ali, SJ Forbes, C Coutelle, N Wright, M Alison, AJ Thrasher, D Bonnet and M Themis Gene Ther. 2006; 13 (2): 117-126

Variable phenotypes in patients diagnosed with idiopathic multifocal choroiditis.
RE MacLaren and SL Lightman Clin Experiment Ophthalmol. 2006; 34 (3): 233-8

Stable and efficient intraocular gene transfer using pseudotyped EIAV lentiviral vectors.
KS Balaggan, K Binley, M Esapa, S Iqball, Z Askham, O Kan, M Tschernutter, JWB Bainbridge, S Naylor and RR Ali J. Gene Med. 2006; 8 (3): 275-285

Absence of Chx10 causes neural progenitors to persist in the adult retina.
NS Dhomen, KS Balaggan, RA Pearson, JW Bainbridge, EM Levine, RR Ali and JC Sowden Invest. Ophthalmol. Vis. Sci. 2006;47 (1): 386-396.

2005


Long-term results of submacular surgery combined with macular translocation of the retinal pigment epithelium in neovascular age-related macular degeneration.
RE MacLaren, AC Bird, PJ Sathia and GW Aylward. Ophthalmology. 2005; 112 (12): 2081-7 

Gene Replacement Therapy Rescues Photoreceptor Degeneration in a Murine Model of Leber Congenital Amaurosis Lacking RPGRIP.
BS Pawlyk, AJ Smith, PK Buch, M Adamian, D Hong, MA Sandberg, RR Ali, and T Li Invest Ophthalmol. Vis. Sci. 2005; 46 (9): 3039-3045

Induction of replication in human corneal endothelial cells by E2F2 transcription factor cDNA transfer.
JC McAlister, NC Joyce, DL Harris, RR Ali and DFP Larkin. Invest. Ophthalmol. Vis. Sci. 2005; 46 (10): 3597-3603.

Local administration of an adeno-associated viral vector expressing IL-10 reduces monocyte infiltration and subsequent photoreceptor damage during experimental autoimmune uveitis.
CA Broderick, AJ Smith, KS Balaggan, A Georgiadis, PK Buch, PC Trittibach, SE Barker, G Sarra, AJ Thrasher, AD Dick and RR Ali. Mol. Ther. 2005; 12 (2): 369-373

Risks of gene therapy should be weighed against lack of alternatives for many diseases.
R Maclaren, RR Ali and AJ Thrasher. BMJ 2005; 330 (7494): 791

Effect of Steroidal and Non-Steroidal Drugs on the Microglia Activation Pattern and the Course of Degeneration in the Retinal Degeneration Slow Mouse.
GM Sarra, FG Sarra, FC Schlichtenbrede, P Trittibach, S Estermann, E Tsiroukis, RR Ali, PJ Luthert and MB Reichel Ophthalmic Res. 2005; 37 (2): 72-82

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

Biometry accuracy using zero- and negative-powered intraocular lenses.
RE MacLaren, MS Sagoo, M Restori and BD Allan J Cataract Refract Surg. 2005; 31 (2): 280-90

Retinal ganglion cell apoptosis in glaucoma is related to intraocular pressure and IOP-induced effects on extracellular matrix.
L Guo, SE Moss, RA Alexander, RR Ali, F Fitzke and MF Cordeiro Invest. Ophthalmol. Vis. Sci. 2005; 46 (1):175-182 

Delayed atrophy of the retinal pigment epithelium after submacular surgery.
RE MacLaren and GW Aylward Eur J Ophthalmol. 2005; 15 (1): 170-2

2004


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

Prospects for gene therapy.
RR Ali Novartis Found. Symp. 2004; 255: 165-72; discussion 173-178. Review

Improvement of neuronal visual responses in the superior colliculus in Prph2(Rd2/Rd2) mice following gene therapy.
FC Schlichtenbrede, AJ Smith, JW Bainbridge, AJ Thrasher, TE Salt and RR Ali Mol. Cell Neurosci. 2004; 25 (1):103-110

Minocycline delays photoreceptor death in the rds mouse through a microglia-independent mechanism.
EH Hughes, FC Schlichtenbrede, CC Murphy, C Broderick, N van Rooijen, RR Ali and AD Dick Exp. Eye Res. 2004; 78 (6):1077-1084

Effect of overexpressing the transcription factor E2F2 on cell cycle progression in rabbit corneal endothelial cells.
NC Joyce, DL Harris, JC McAlister, RR Ali and DF Larkin. Invest. Ophthalmol. Vis. Sci.2004; 45 (5):1340-1348

Permanent phenotypic correction of hemophilia B in immunocompetent mice by prenatal gene therapy.
SN Waddington, MS Nivsarkar, AR Mistry, SM Buckley, G Kemball-Cook, KL Mosley, K Mitrophanous, P Radcliffe, MV Holder, M Brittan, A Georgiadis, F Al-Allaf, BW Bigger, LG Gregory, HT Cook, RR Ali, A Thrasher, EG Tuddenham, M Themis and C Coutelle Blood 2004; 104 (9): 2714-2721 

Marked inhibition of retinal neovascularization in rats following soluble-flt-1 gene transfer.
R Rota, T Riccioni, M Zaccarini, S Lamartina, AD Gallo, A Fusco, I Kovesdi, E Balestrazzi, DC Abeni, RR Ali and MC Capogrossi. J. Gene Med. 2004; 6 (9): 992-1002

2003


Therapy may yet stem from cells in the retina.
Ali RR and JC Sowden Br. J. Ophthalmol. 2003; 87 (9):1058-1059 

Longterm evaluation of retinal function in Prph2Rd2/Rd2 mice following AAV-mediated gene replacement therapy.
FC Schlichtenbrede, L daCruz, C Stephens, AJ Smith, A Geogiardis, AJ Thrasher, JWB Bainbridge, M Seeliger and RR Ali J. Gene Med. 2003; 5 (9): 757-764

Gene therapy for ocular angiogenesis.
JW Bainbridge, A Mistry, AJ Thrasher and RR Ali Clin. Sci. 2003; 104 (6): 561-575

A peptide encoded by exon 6 of VEGF (EG3306) inhibits VEGF-induced angiogenesis in vitro and ischaemic retinal neovascularisation in vivo.
JWB Bainbridge, H Jia, A Bagherzadeh, D Selwood, RR Ali and I Zachary Biochem. Biophys. Res. Commun. 2003; 302 (4): 793-799

AAV-mediated gene transfer slows photoreceptor loss in the RCS rat model of retinitis pigmentosa.
AJ Smith, FC Schlichtenbrede, M Tschernutter, JW Bainbridge, AJ Thrasher and RR Ali Mol. Ther. 2003;8 (2): 118-195

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

Hypoxia-regulated transgene expression in experimental retinal and choroidal neovascularisation.
JWB Bainbridge, A Mistry, K Binley, M De Alwis, AJ Thrasher, Susan Kingsman, S Naylor and RR AliGene Ther. 2003; 10: 1049-1054

Generation of activated sialoadhesin-positive microglia during retinal degeneration.
EH Hughes, F Schlichtenbrede, CC Murphy, G-M Sarra, P Luthert, RR Ali and AD Dick Invest. Ophthalmol. Vis. Sci.2003; 44 (5): 2229-2234

Intraocular gene delivery of ciliary neurotrophic factor results in significant loss of retinal function in normal mice and in the Prph2Rd2/Rd2 model for retinal degenration.
FC Schlichtenbrede, A McNeil, AJ Smith, M Tschernutter, JWB Bainbridge and RR Ali Gene Ther. 2003; 10 (6): 523-527

Novel antisense oligonucleotides targeting TGF-ß inhibit in vivo scarring and improve surgical outcome.
MF Cordeiro, A Mead, RR Ali, RA Alexander, S Murray, C Chen, C York-Defalco, NM Dean, GS Schultz and PT Khaw. Gene Ther. 2003; 10 (1): 59-71

2002


Long-term reversal of chronic anemia using a hypoxia-regulated erythropoietin gene therapy.
K Binley, Z Askham, S Iqball, H Spearman, L Martin, M De Alwis, AJ Thrasher, RR Ali, PH Maxwell, S Kingsman and S Naylor Blood 2002; 100 (7): 2406-2413.

High titre stocks of adeno-associated virus from replicating amplicons and herpes virus.
A Mistry, M De Alwis, E Feudner, RR Ali and AJ Thrasher Methods Mol. Med. 2002; 69: 445-460

Inhibition of retinal neovascularisation by gene transfer of soluble VEGF receptor sFlt-1.
JWB Bainbridge, A Mistry, M De Alwis, E Paleolog, A Baker, AJ Thrasher and RR Ali Gene Ther. 2002; 9 (5): 320-326

Kinetics of transgene expression in mouse retina following sub-retinal injection of recombinant adeno-associated virus.
G Sarra, C Stephens, F Schlichtenbrede, JWB Bainbridge, AJ Thrasher, PJ Luthert and RR Ali Vision Res. 2002; 42 (4): 541-549

Spontaneous foreign body extrusion following perforating eye injury.
RR Bourne, M Parulekar, AS Bacon and RE MacLaren J R Army Med Corps. 2002; 148 (2): 144-7

[Progress in somatic gene therapy of retinal degeneration in the animal model].
FC Schlichtenbrede, GM Sarra, RR Ali, P Wiedemann and MB Reichel Ophthalmologe 2002; 99 (4): 259-265

Benign idiopathic haemorrhagic retinopathy.
RE MacLaren Eye. 2002; 16 (1): 107-8

2001


Molecular genetics and prospects for therapy of the inherited retinal dystrophies.
DA Bessant, RR Ali and SS Bhattacharya. Curr. Opin. Genet. Dev. 2001; 11 (3): 307-316

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

Efficient and Selective AAV2-Mediated Gene Transfer Directed to Human Vascular Endothelial Cells.
SA Nicklin, H Buening, K Dishart, M De Alwis, A Girod, U Hacker, AJ Thrasher, RR Ali, M Hallek and AH Baker. Mol. Ther. 2001; 4 (3): 174-181

Gene replacement therapy in the retinal degeneration slow (rds) mouse: the effect on retinal degeneration following a partial transduction of the retina.
G Sarra, C Stephens, M de Alwis, JWB Bainbridge, AJ Smith, AJ Thrasher and RR Ali Hum. Mol. Genet. 2001; 10 (21): 2353-2361

Optimization of recombinant adeno-associated virus production using an herpes simplex virus amplicon system.
E Feudner, M De Alwis, AJ Thrasher, RR Ali, and S Fauser J. Virol. Methods 2001; 96 (2): 97-105.

An immune response after intraocular administration of an adenoviral vector containing a beta galactosidase reporter gene slows retinal degeneration in the rd mouse.
MB Reichel, JWB Bainbridge, D Baker, AJ Thrasher, SS Bhattacharya and RR Ali Br. J. Ophthalmol. 2001; 85 (3): 341-344

2000


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

Fluorescein angiography in altitude retinopathy.
RE MacLaren, K Ikram and SJ Talks Br J Ophthalmol. 2000; 84 (3): 339-400

Decreased visual acuity in patients with cytomegalovirus retinitis and AIDS.
J Raina, JW Bainbridge and SM Shah Eye. 2000; 14 ( Pt 1): 8-12

Details of publications published prior to 2000 can be found on Pub Med

Page last modified on 20 jun 13 14:31


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