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Expert Opinion on Biological Therapy Volume 20, 2020 - Issue 1
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Review

RPGR gene therapy presents challenges in cloning the coding sequence

Cristina Martinez-Fernandez De La CamaraDepartment of Clinical Neurosciences, Nuffield Laboratory of Ophthalmology, University of Oxford, John Radcliffe Hospital, Headley Way, UK;Oxford Eye Hospital, Oxford University Hospitals NHS Trust, John Radcliffe Hospital, Headley Way, UKView further author information
,
Jasmina Cehajic-KapetanovicDepartment of Clinical Neurosciences, Nuffield Laboratory of Ophthalmology, University of Oxford, John Radcliffe Hospital, Headley Way, UK;Oxford Eye Hospital, Oxford University Hospitals NHS Trust, John Radcliffe Hospital, Headley Way, UKView further author information
&
Robert E. MacLarenDepartment of Clinical Neurosciences, Nuffield Laboratory of Ophthalmology, University of Oxford, John Radcliffe Hospital, Headley Way, UK;Oxford Eye Hospital, Oxford University Hospitals NHS Trust, John Radcliffe Hospital, Headley Way, UKCorrespondence[email protected]
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Pages 63-71 | Received 05 Aug 2019, Accepted 11 Oct 2019, Published online: 20 Oct 2019

Citations (8)

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Nataša Mihailovic, Simone Schimpf-Linzenbold, Inga Sattler, Nicole Eter & Peter Heiduschka. (2022) The first reported case of a deletion of the entire RPGR gene in a family with X-linked retinitis pigmentosa. Ophthalmic Genetics 43:5, pages 679-684.
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Articles from other publishers (7)

Lenore von Krusenstiern, Jiajun Liu, Eileen Liao, James A. Gow, Guo Chen, Tuyen Ong, Andrew J. Lotery, Assad Jalil, Byron L. Lam, Robert E. MacLaren, Imran H. Yusuf, Jasmina Cehajic-Kapetanovic, Amandeep S. Josan, Cristina Martinez-Fernandez de la Camera, Salwah  Rehman, Laura J. Wood, Jasleen K. Jolly, Kanmin  Xue, Anika Nanda, Kirti Jasani, Moreno  Menghini, Thomas M.W. Buckley, Anna Paola  Salvetti, Suresh  Thulasidharan, Miguel  Kurc, Samir Khandhadia, Karla Orsine Murta Dias, Abeir  Baltmr, Nakul Mandal, Georgios  Tsokolas, Paulo Stanga, Tsveta Ivanova, Muhannd EL-Faouri, Sherif  Shaarawy, Graeme Black, Janet Louise Davis, Ninel Gregori, Carlos E.  Mendoza-Santiesteban, Potyra R. Rosa, Kevin G. Evans, Rob Koenekoop, Dominik Fischer, Frank Holz, Kamron Khan, Jason Horowitz, Mark Pennesi, David Birch, Michael Gorin, Kim Stepien, Jacque Duncan, Tim Stout, Benjamin Bakall, Paul S. Bernstein, Eeva-Maria Sankila, Carel Hoyng, Camiel Boon & Isabelle Meunier. (2023) Changes in Retinal Sensitivity Associated With Cotoretigene Toliparvovec in X-Linked Retinitis Pigmentosa With RPGR Gene Variations . JAMA Ophthalmology 141:3, pages 275.
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Vahid Mansouri. (2022) X-Linked Retinitis Pigmentosa Gene Therapy: Preclinical Aspects. Ophthalmology and Therapy 12:1, pages 7-34.
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Jessica D. Gumerson, Amal Alsufyani, Wenhan Yu, Jingqi Lei, Xun Sun, Lijin Dong, Zhijian Wu & Tiansen Li. (2021) Restoration of RPGR expression in vivo using CRISPR/Cas9 gene editing. Gene Therapy 29:1-2, pages 81-93.
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Aumer Shughoury, Thomas A. Ciulla, Benjamin Bakall, Mark E. Pennesi, Szilárd Kiss & Emmett T. Cunningham. (2021) Genes and Gene Therapy in Inherited Retinal Disease. International Ophthalmology Clinics 61:4, pages 3-45.
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Jasmina Cehajic-Kapetanovic, Michelle E. McClements, Jennifer Whitfield, Morag Shanks, Penny Clouston & Robert E. MacLaren. (2020) Association of a Novel Intronic Variant in RPGR With Hypomorphic Phenotype of X-Linked Retinitis Pigmentosa . JAMA Ophthalmology 138:11, pages 1151.
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Francesco Parmeggiani. (2020) X-Chromosome Insight for Targeting Gene Therapy. Ophthalmology Retina 4:5, pages 521-522.
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Emmanuelle Clérin, Myriam Marussig, José-Alain Sahel & Thierry Léveillard. (2020) Metabolic and Redox Signaling of the Nucleoredoxin-Like-1 Gene for the Treatment of Genetic Retinal Diseases. International Journal of Molecular Sciences 21:5, pages 1625.
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