Browse by UCL people
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Number of items: 42.
Article
Aguilà, M;
Bellingham, J;
Athanasiou, D;
Bevilacqua, D;
Duran, Y;
Maswood, R;
Parfitt, DA;
... Cheetham, ME; + view all
(2020)
AAV-mediated ERdj5 overexpression protects against P23H rhodopsin toxicity.
Human Molecular Genetics
, 29
(8)
pp. 1310-1318.
10.1093/hmg/ddaa049.
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Auricchio, A;
Smith, AJ;
Ali, RR;
(2017)
The Future Looks Brighter After 25 Years of Retinal Gene Therapy.
Human Gene Therapy
, 28
(11)
pp. 982-987.
10.1089/hum.2017.164.
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Bainbridge, JWB;
Mehat, MS;
Sundaram, V;
Robbie, SJ;
Barker, SE;
Ripamonti, C;
Georgiadis, A;
... Ali, RR; + view all
(2015)
Long-Term Effect of Gene Therapy on Leber's Congenital Amaurosis.
New England Journal of Medicine
, 372
(20)
pp. 1887-1897.
10.1056/NEJMoa1414221.
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Basche, M;
Kampik, D;
Kawasaki, S;
Branch, MJ;
Robinson, M;
Larkin, DF;
Smith, AJ;
(2018)
Sustained and Widespread Gene Delivery to the Corneal Epithelium via in Situ Transduction of Limbal Epithelial Stem Cells, Using Lentiviral and Adeno-associated Viral Vectors.
Human Gene Therapy
, 29
(10)
pp. 1140-1152.
10.1089/hum.2018.115.
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Carvalho, LS;
Xu, JH;
Pearson, RA;
Smith, AJ;
Bainbridge, JW;
Morris, LM;
Fliesler, SJ;
... Ali, RR; + view all
(2011)
Long-term and age-dependent restoration of visual function in a mouse model of CNGB3-associated achromatopsia following gene therapy.
Human Molecular Genetics
, 20
(16)
3161 - 3175.
10.1093/hmg/ddr218.
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Chu, CJ;
Gardner, PJ;
Copland, DA;
Liyanage, SE;
Gonzalez-Cordero, A;
Kleine Holthaus, SM;
Luhmann, UF;
... Dick, AD; + view all
(2016)
Multimodal analysis of ocular inflammation using the endotoxin-induced uveitis mouse model.
Disease Models and Mechanisms
, 9
(4)
pp. 473-481.
10.1242/dmm.022475.
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Cristante, E;
Liyanage, SE;
Sampson, RD;
Kalargyrou, A;
De Rossi, G;
Rizzi, M;
Hoke, J;
... Bainbridge, JWB; + view all
(2018)
Late neuroprogenitors contribute to normal retinal vascular development in aHif2a-dependent manner.
Development
, 145
(8)
, Article dev157511. 10.1242/dev.157511.
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Feathers, KL;
Jia, L;
Perera, ND;
Chen, A;
Presswalla, FK;
Khan, NW;
Fahim, AT;
... Thompson, DA; + view all
(2019)
Development of a Gene Therapy Vector for RDH12-Associated Retinal Dystrophy.
Human Gene Therapy
, 30
(11)
pp. 1325-1335.
10.1089/hum.2019.017.
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Georgiadis, A;
Duran, Y;
Ribeiro, J;
Abelleira-Hervas, L;
Robbie, SJ;
Sünkel-Laing, B;
Fourali, S;
... Ali, RR; + view all
(2016)
Development of an optimized AAV2/5 gene therapy vector for Leber congenital amaurosis owing to defects in RPE65.
Gene Therapy
, 23
(12)
pp. 857-862.
10.1038/gt.2016.66.
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Gonzalez Cordero, A;
Goh, D;
Kruczek, K;
Naeem, A;
Fernando, M;
Kleine Holthaus, S-M;
Takaaki, M;
... Ali, RR; + view all
(2018)
Assessment of AAV vector tropisms for mouse and human pluripotent stem cell-derived RPE and photoreceptor cells.
Human Gene Therapy
, 29
(10)
pp. 1124-1139.
10.1089/hum.2018.027.
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Gonzalez-Cordero, A;
Kruczek, K;
Naeem, A;
Fernando, M;
Kloc, M;
Ribeiro, J;
Goh, D;
... Ali, RR; + view all
(2017)
Recapitulation of Human Retinal Development from Human Pluripotent Stem Cells Generates Transplantable Populations of Cone Photoreceptors.
Stem Cell Reports
10.1016/j.stemcr.2017.07.022.
(In press).
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Hippert, C;
Graca, AB;
Barber, AC;
West, EL;
Smith, AJ;
Ali, RR;
Pearson, RA;
(2015)
Müller glia activation in response to inherited retinal degeneration is highly varied and disease-specific.
PLoS One
, 10
(3)
, Article e0120415. 10.1371/journal.pone.0120415.
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Holthaus, S-MK;
Herranz-Martin, S;
Massaro, G;
Aristorena, M;
Hoke, J;
Hughes, MP;
Maswood, R;
... Ali, RR; + view all
(2019)
Neonatal brain-directed gene therapy rescues a mouse model of neurodegenerative CLN6 Batten disease.
Human Molecular Genetics
, 28
(23)
pp. 3867-3879.
10.1093/hmg/ddz210.
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Kampik, D;
Basche, M;
Georgiadis, A;
Luhmann, UFO;
Larkin, DF;
Smith, AJ;
Ali, RR;
(2019)
Modulation of Contact Inhibition by ZO-1/ZONAB Gene Transfer-A New Strategy to Increase the Endothelial Cell Density of Corneal Grafts.
Investigative Ophthalmology & Visual Science
, 60
(8)
pp. 3170-3177.
10.1167/iovs.18-26260.
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Kampik, D;
Basche, M;
Luhmann, UFO;
Nishiguchi, KM;
Williams, JAE;
Greenwood, J;
Moss, SE;
... Ali, RR; + view all
(2017)
In situ regeneration of retinal pigment epithelium by gene transfer of E2F2: a potential strategy for treatment of macular degenerations.
Gene Therapy
, 24
pp. 810-818.
10.1038/gt.2017.89.
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Kleine Holthaus, S-M;
Aristorena, M;
Maswood, R;
Semenyuk, O;
Hoke, J;
Hare, A;
Smith, AJ;
... Ali, RR; + view all
(2020)
Gene Therapy Targeting the Inner Retina Rescues the Retinal Phenotype in a Mouse Model of CLN3 Batten Disease.
Human Gene Therapy
, 31
(13-14)
pp. 709-718.
10.1089/hum.2020.038.
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Kleine Holthaus, S-M;
Ribeiro, J;
Abelleira-Hervas, L;
Pearson, RA;
Duran, Y;
Georgiadis, A;
Sampson, RD;
... Ali, RR; + view all
(2018)
Prevention of Photoreceptor Cell Loss in a Cln6nclf Mouse Model of Batten Disease Requires CLN6 Gene Transfer to Bipolar Cells.
Molecular Therapy
, 26
(5)
pp. 1343-1353.
10.1016/j.ymthe.2018.02.027.
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Kruczek, K;
Gonzalez-Cordero, A;
Goh, D;
Naeem, A;
Jonikas, M;
Blackford, SJI;
Kloc, M;
... Ali, RR; + view all
(2017)
Differentiation and Transplantation of Embryonic Stem Cell-Derived Cone Photoreceptors into a Mouse Model of End-Stage Retinal Degeneration.
Stem Cell Reports
, 8
(6)
pp. 1659-1674.
10.1016/j.stemcr.2017.04.030.
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Kumaran, N;
Michaelides, M;
Smith, AJ;
Ali, RR;
Bainbridge, JWB;
(2018)
Retinal gene therapy.
British Medical Bulletin
, 126
(1)
pp. 13-25.
10.1093/bmb/ldy005.
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Kumaran, N;
Smith, AJ;
Michaelides, M;
Ali, R;
Bainbridge, J;
(2018)
Gene therapy for Leber congenital amaurosis.
[Review].
Expert Review of Ophthalmology
, 13
(1)
pp. 11-15.
10.1080/17469899.2018.1429916.
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Lange, C;
Mowat, F;
Sayed, H;
Mehad, M;
Duluc, L;
Piper, S;
Luhmann, U;
... Bainbridge, J; + view all
(2016)
Dimethylarginine dimethylaminohydrolase-2 deficiency promotes vascular regeneration and attenuates pathological angiogenesis.
Experimental Eye Research
, 147
pp. 148-155.
10.1016/j.exer.2016.05.007.
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Liu, W;
Kleine-Holthaus, S-M;
Herranz-Martin, S;
Aristorena, M;
Mole, SE;
Smith, AJ;
Ali, RR;
(2020)
Experimental gene therapies for the NCLs.
Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease
, 1866
(9)
, Article 165772. 10.1016/j.bbadis.2020.165772.
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Liyanage, SE;
Fantin, A;
Villacampa, P;
Lange, CA;
Denti, L;
Cristante, E;
Smith, AJ;
... Ruhrberg, C; + view all
(2016)
Myeloid-Derived Vascular Endothelial Growth Factor and Hypoxia-Inducible Factor Are Dispensable for Ocular Neovascularization-Brief Report.
Arteriosclerosis, Thrombosis, and Vascular Biology
, 36
(1)
pp. 19-24.
10.1161/ATVBAHA.115.306681.
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Luhmann, UF;
Carvalho, LS;
Holthaus, SM;
Cowing, JA;
Greenaway, S;
Chu, CJ;
Herrmann, P;
... Ali, RR; + view all
(2015)
The severity of retinal pathology in homozygous Crb1rd8/rd8 mice is dependent on additional genetic factors.
Hum Mol Genet
, 24
(1)
128 - 141.
10.1093/hmg/ddu424.
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Mehat, MS;
Sundaram, V;
Ripamonti, C;
Robson, AG;
Smith, AJ;
Borooah, S;
Robinson, M;
... Bainbridge, JWB; + view all
(2018)
Transplantation of Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial Cells in Macular Degeneration.
Ophthalmology
, 125
(11)
pp. 1765-1775.
10.1016/j.ophtha.2018.04.037.
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Mihelec, M;
Pearson, RA;
Robbie, SJ;
Buch, PK;
Azam, SA;
Bainbridge, JWB;
Smith, AJ;
(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.
Human Gene Therapy
, 22
(10)
1179 - 1190.
10.1089/hum.2011.069.
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Mole, SE;
Anderson, G;
Band, HA;
Berkovic, SF;
Cooper, JD;
Kleine Holthaus, S-M;
McKay, TR;
... Smith, AJ; + view all
(2019)
Clinical challenges and future therapeutic approaches for neuronal ceroid lipofuscinosis.
[Review].
Lancet Neurology
, 18
(1)
pp. 107-116.
10.1016/S1474-4422(18)30368-5.
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Mowat, FM;
Gervais, KJ;
Occelli, LM;
Annear, MJ;
Querubin, J;
Bainbridge, JW;
Smith, AJ;
... Petersen-Jones, SM; + view all
(2017)
Early-Onset Progressive Degeneration of the Area Centralis in RPE65-Deficient Dogs.
Invest Ophthalmol Vis Sci
, 58
(7)
pp. 3268-3277.
10.1167/iovs.17-21930.
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Mowat, FM;
Luhmann, UFO;
Smith, AJ;
Lange, C;
Duran, Y;
Harten, S;
Shukla, D;
... Bainbridge, JWB; + view all
(2010)
HIF-1alpha and HIF-2alpha Are Differentially Activated in Distinct Cell Populations in Retinal Ischaemia.
PLOS ONE
, 5
(6)
, Article e11103. 10.1371/journal.pone.0011103.
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Nishiguchi, KM;
Carvalho, LS;
Rizzi, M;
Powell, K;
Holthaus, SM;
Azam, SA;
Duran, Y;
... Ali, RR; + view all
(2015)
Gene therapy restores vision in rd1 mice after removal of a confounding mutation in Gpr179.
Nature Communications
, 6
, Article 6606. 10.1038/ncomms7006.
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Ovando-Roche, P;
Georgiadis, A;
Smith, AJ;
Pearson, RA;
Ali, RR;
(2017)
Harnessing the Potential of Human Pluripotent Stem Cells and Gene Editing for the Treatment of Retinal Degeneration.
Current Stem Cell Reports
, 3
(2)
pp. 112-123.
10.1007/s40778-017-0078-4.
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Ovando-Roche, P;
West, EL;
Branch, MJ;
Sampson, RD;
Fernando, M;
Munro, P;
Georgiadis, A;
... Ali, RR; + view all
(2018)
Use of bioreactors for culturing human retinal organoids improves photoreceptor yields.
Stem Cell Research & Therapy
, 9
(1)
, Article 156. 10.1186/s13287-018-0907-0.
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Pearson, RA;
Gonzalez-Cordero, A;
West, EL;
Ribeiro, JR;
Aghaizu, N;
Goh, D;
Sampson, RD;
... Ali, RR; + view all
(2016)
Donor and host photoreceptors engage in material transfer following transplantation of post-mitotic photoreceptor precursors.
Nature Communications
, 7
, Article 13029. 10.1038/ncomms13029.
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Tan, M. H.;
Smith, A. J.;
Pawlyk, B.;
Xu, X.;
Liu, X.;
Bainbridge, J. B.;
Basche, M.;
... Ali, R. R.; + view all
(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.
Human Molecular Genetics
, 18
(12)
pp. 2099-2114.
10.1093/hmg/ddp133.
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Tan, MH;
Mackay, DS;
Cowing, J;
Tran, HV;
Smith, AJ;
Wright, GA;
Dev-Borman, A;
... Moore, AT; + view all
(2012)
Leber congenital amaurosis associated with AIPL1: Challenges in ascribing disease causation, clinical findings, and implications for gene therapy.
PLoS ONE
, 7
(3)
10.1371/journal.pone.0032330.
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Tan, MH;
Mackay, DS;
Cowing, J;
Tran, HV;
Smith, AJ;
Wright, GA;
Dev-Borman, A;
... Moore, AT; + view all
(2012)
Leber congenital amaurosis associated with AIPL1: challenges in ascribing disease causation, clinical findings, and implications for gene therapy.
PLoS One
, 7
(3)
, Article e32330. 10.1371/journal.pone.0032330.
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Tan, MH;
Smith, AJ;
Pawlyk, B;
Xu, XY;
Liu, XQ;
Bainbridge, JB;
Basche, M;
... Ali, RR; + view all
(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.
Human Molecular Genetics
, 18
(12)
2099 - 2114.
10.1093/hmg/ddp133.
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Tee, JJ;
Smith, AJ;
Hardcastle, AJ;
Michaelides, M;
(2016)
RPGR-associated retinopathy: clinical features, molecular genetics, animal models and therapeutic options.
British Journal of Ophthalmology
, 100
(8)
pp. 1022-1027.
10.1136/bjophthalmol-2015-307698.
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Villacampa, P;
Liyanage, SE;
Klaska, IP;
Cristante, E;
Menger, KE;
Sampson, RD;
Barlow, M;
... Bainbridge, JWB; + view all
(2019)
Stabilization of myeloid-derived HIFs promotes vascular regeneration in retinal ischemia.
Angiogenesis
10.1007/s10456-019-09681-1.
(In press).
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Villacampa, P;
Menger, KE;
Abelleira, L;
Ribeiro, J;
Duran, Y;
Smith, AJ;
Ali, RR;
... Bainbridge, JWB; + view all
(2017)
Accelerated oxygen-induced retinopathy is a reliable model of ischemia-induced retinal neovascularization.
PLoS ONE
, 12
(6)
, Article e0179759. 10.1371/journal.pone.0179759.
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Waldron, PV;
Di Marco, F;
Kruczek, K;
Ribeiro, J;
Graca, AB;
Hippert, C;
Aghaizu, ND;
... Pearson, RA; + view all
(2018)
Transplanted Donor- or Stem Cell-Derived Cone Photoreceptors Can Both Integrate and Undergo Material Transfer in an Environment-Dependent Manner.
Stem Cell Reports
10.1016/j.stemcr.2017.12.008.
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Proceedings paper
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Kleine Holthaus, S-M;
Smith, AJ;
Mole, SE;
Ali, RR;
(2018)
Gene Therapy Approaches to Treat the Neurodegeneration and Visual Failure in Neuronal Ceroid Lipofuscinoses.
In:
Retinal Degenerative Diseases.
(pp. pp. 91-99).
Springer: Cham, Switzerland.
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