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Year Number of Results
1996 1
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1999 3
2000 1
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2003 10
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2005 9
2006 16
2007 8
2008 9
2009 8
2010 11
2011 16
2012 21
2013 12
2014 9
2015 14
2016 11
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2019 8
2020 12
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274 results

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Page 1
Retinal gene therapy.
Kumaran N, Michaelides M, Smith AJ, Ali RR, Bainbridge JWB. Kumaran N, et al. Among authors: ali rr. Br Med Bull. 2018 Jun 1;126(1):13-25. doi: 10.1093/bmb/ldy005. Br Med Bull. 2018. PMID: 29506236 Review.
Phase 1/2 AAV5-hRKp.RPGR (Botaretigene Sparoparvovec) Gene Therapy: Safety and Efficacy in RPGR-Associated X-Linked Retinitis Pigmentosa.
Michaelides M, Besirli CG, Yang Y, DE Guimaraes TAC, Wong SC, Huckfeldt RM, Comander JI, Sahel JA, Shah SM, Tee JJL, Kumaran N, Georgiadis A, Minnick P, Zeldin R, Naylor S, Xu J, Clark M, Anglade E, Wong P, Fleck PR, Fung A, Peluso C, Kalitzeos A, Georgiou M, Ripamonti C, Smith AJ, Ali RR, Forbes A, Bainbridge J. Michaelides M, et al. Among authors: ali rr. Am J Ophthalmol. 2024 Nov;267:122-134. doi: 10.1016/j.ajo.2024.05.034. Epub 2024 Jun 12. Am J Ophthalmol. 2024. PMID: 38871269 Free article. Clinical Trial.
Gene therapy in children with AIPL1-associated severe retinal dystrophy: an open-label, first-in-human interventional study.
Michaelides M, Laich Y, Wong SC, Oluonye N, Zaman S, Kumaran N, Kalitzeos A, Petrushkin H, Georgiou M, Tailor V, Pabst M, Staeubli K, Maimon-Mor RO, Jones PR, Scholte SH, Georgiadis A, van der Spuy J, Naylor S, Forbes A, Dekker TM, Arulmuthu ER, Smith AJ, Ali RR, Bainbridge JWB. Michaelides M, et al. Among authors: ali rr. Lancet. 2025 Feb 22;405(10479):648-657. doi: 10.1016/S0140-6736(24)02812-5. Lancet. 2025. PMID: 39986747 Free article.
Experimental gene therapies for the NCLs.
Liu W, Kleine-Holthaus SM, Herranz-Martin S, Aristorena M, Mole SE, Smith AJ, Ali RR, Rahim AA. Liu W, et al. Among authors: ali rr. Biochim Biophys Acta Mol Basis Dis. 2020 Sep 1;1866(9):165772. doi: 10.1016/j.bbadis.2020.165772. Epub 2020 Mar 24. Biochim Biophys Acta Mol Basis Dis. 2020. PMID: 32220628 Free article. Review.
Advancing therapeutic strategies for inherited retinal degeneration: recommendations from the Monaciano Symposium.
Thompson DA, Ali RR, Banin E, Branham KE, Flannery JG, Gamm DM, Hauswirth WW, Heckenlively JR, Iannaccone A, Jayasundera KT, Khan NW, Molday RS, Pennesi ME, Reh TA, Weleber RG, Zacks DN; Monaciano Consortium. Thompson DA, et al. Among authors: ali rr. Invest Ophthalmol Vis Sci. 2015 Feb 9;56(2):918-31. doi: 10.1167/iovs.14-16049. Invest Ophthalmol Vis Sci. 2015. PMID: 25667399 Free PMC article. Review.
Human cone photoreceptor transplantation stimulates remodeling and restores function in AIPL1 model of end-stage Leber congenital amaurosis.
Procyk CA, Melati A, Ribeiro J, Liu J, Branch MJ, Delicata JD, Tariq M, Kalarygrou AA, Kapadia J, Khorsani MM, West EL, Smith AJ, Gonzalez-Cordero A, Ali RR, Pearson RA. Procyk CA, et al. Among authors: ali rr. Stem Cell Reports. 2025 Apr 8;20(4):102470. doi: 10.1016/j.stemcr.2025.102470. Epub 2025 Mar 27. Stem Cell Reports. 2025. PMID: 40154478 Free PMC article.
PRPF8-mediated dysregulation of hBrr2 helicase disrupts human spliceosome kinetics and 5´-splice-site selection causing tissue-specific defects.
Atkinson R, Georgiou M, Yang C, Szymanska K, Lahat A, Vasconcelos EJR, Ji Y, Moya Molina M, Collin J, Queen R, Dorgau B, Watson A, Kurzawa-Akanbi M, Laws R, Saxena A, Shyan Beh C, Siachisumo C, Goertler F, Karwatka M, Davey T, Inglehearn CF, McKibbin M, Lührmann R, Steel DH, Elliott DJ, Armstrong L, Urlaub H, Ali RR, Grellscheid SN, Johnson CA, Mozaffari-Jovin S, Lako M. Atkinson R, et al. Among authors: ali rr. Nat Commun. 2024 Apr 11;15(1):3138. doi: 10.1038/s41467-024-47253-0. Nat Commun. 2024. PMID: 38605034 Free PMC article.
LRG1 Alters Pericyte Phenotype and Compromises Vascular Maturation.
Hoeh AE, Chang JH, Mueller RS, Basche M, Fantin A, Sepetis A, De Rossi G, Dritsoula A, Ali RR, Turowski P, Moss SE, Greenwood J. Hoeh AE, et al. Among authors: ali rr. Cells. 2025 Apr 14;14(8):593. doi: 10.3390/cells14080593. Cells. 2025. PMID: 40277918 Free PMC article.
Gene therapy for noninfectious uveitis.
Chu CJ, Barker SE, Dick AD, Ali RR. Chu CJ, et al. Among authors: ali rr. Ocul Immunol Inflamm. 2012 Dec;20(6):394-405. doi: 10.3109/09273948.2012.726392. Epub 2012 Nov 19. Ocul Immunol Inflamm. 2012. PMID: 23163799 Review.
274 results