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Expert Opinion on Biological Therapy Volume 18, 2018 - Issue 7
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Review

Choroideremia: molecular mechanisms and development of AAV gene therapy

Maria I PatrícioNuffield Laboratory of Ophthalmology, Nuffield Department of Clinical Neurosciences, University of Oxford, Oxford, UK;National Institute for Health Research (NIHR) Oxford Biomedical Research Centre (BRC), Oxford, UK;Oxford Eye Hospital, Oxford University Hospitals NHS Foundation Trust, Oxford, UKView further author information
,
Alun R BarnardNuffield Laboratory of Ophthalmology, Nuffield Department of Clinical Neurosciences, University of Oxford, Oxford, UK;National Institute for Health Research (NIHR) Oxford Biomedical Research Centre (BRC), Oxford, UK;Oxford Eye Hospital, Oxford University Hospitals NHS Foundation Trust, Oxford, UKView further author information
,
Kanmin XueNuffield Laboratory of Ophthalmology, Nuffield Department of Clinical Neurosciences, University of Oxford, Oxford, UK;National Institute for Health Research (NIHR) Oxford Biomedical Research Centre (BRC), Oxford, UK;Oxford Eye Hospital, Oxford University Hospitals NHS Foundation Trust, Oxford, UKView further author information
&
Robert E MacLarenNuffield Laboratory of Ophthalmology, Nuffield Department of Clinical Neurosciences, University of Oxford, Oxford, UK;National Institute for Health Research (NIHR) Oxford Biomedical Research Centre (BRC), Oxford, UK;Oxford Eye Hospital, Oxford University Hospitals NHS Foundation Trust, Oxford, UKCorrespondence[email protected]
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Pages 807-820 | Received 13 Feb 2018, Accepted 31 May 2018, Published online: 22 Jun 2018

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Monica L Hu, Thomas L Edwards, Fleur O’Hare, Doron G Hickey, Jiang-Hui Wang, Zhengyang Liu & Lauren N Ayton. (2021) Gene therapy for inherited retinal diseases: progress and possibilities. Clinical and Experimental Optometry 104:4, pages 444-454.
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Articles from other publishers (13)

Samantha R. De Silva, Gavin Arno, Anthony G. Robson, Ana Fakin, Nikolas Pontikos, Moin D. Mohamed, Alan C. Bird, Anthony T. Moore, Michel Michaelides, Andrew R. Webster & Omar A. Mahroo. (2021) The X-linked retinopathies: Physiological insights, pathogenic mechanisms, phenotypic features and novel therapies. Progress in Retinal and Eye Research 82, pages 100898.
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Rocio Maldonado, Sami Jalil & Kirmo Wartiovaara. (2020) Curative gene therapies for rare diseases. Journal of Community Genetics 12:2, pages 267-276.
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Maria Pilar Martin-Gutierrez, Thomas MW Buckley & Robert E MacLaren. (2021) Chronic untreated retinal detachment in a patient with choroideremia provides insight into the disease process and potential therapy. European Journal of Ophthalmology, pages 112067212199472.
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Alessandro Abbouda, Filippo Avogaro, Mariya Moosajee & Enzo Maria Vingolo. (2021) Update on Gene Therapy Clinical Trials for Choroideremia and Potential Experimental Therapies. Medicina 57:1, pages 64.
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Yi Zhai, Manlong Xu, Ioannis S. Dimopoulos, David G. Birch, Paul S. Bernstein, Jenny Holt, David Kirn, Peter Francis & Ian M. MacDonald. (2020) Quantification of RPE Changes in Choroideremia Using a Photoshop-Based Method. Translational Vision Science & Technology 9:7, pages 21.
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Maria I. Patrício, Christopher I. Cox, Clare Blue, Alun R. Barnard, Cristina Martinez-Fernandez de la Camara & Robert E. MacLaren. (2020) Inclusion of PF68 Surfactant Improves Stability of rAAV Titer when Passed through a Surgical Device Used in Retinal Gene Therapy. Molecular Therapy - Methods & Clinical Development 17, pages 99-106.
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Mariana Matioli da Palma, Fabiana Louise Motta, Caio Perez Gomes, Mariana Vallim Salles, João Bosco Pesquero & Juliana Maria Ferraz Sallum. (2020) Synonymous Variant in the CHM Gene Causes Aberrant Splicing in Choroideremia . Investigative Opthalmology & Visual Science 61:2, pages 38.
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Lewis E. Fry, Maria I. Patrício, Jonathan Williams, James W. Aylward, Harriet Hewitt, Penny Clouston, Kanmin Xue, Alun R. Barnard & Robert E. MacLaren. (2020) Association of Messenger RNA Level With Phenotype in Patients With Choroideremia. JAMA Ophthalmology 138:2, pages 128.
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David M. Gamm, Eric Clark, Elizabeth E. Capowski & Ruchira Singh. (2019) The Role of FGF9 in the Production of Neural Retina and RPE in a Pluripotent Stem Cell Model of Early Human Retinal Development. American Journal of Ophthalmology 206, pages 113-131.
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Jasmina Cehajic Kapetanovic, Alun R. Barnard & Robert E. MacLaren. (2019) Molecular Therapies for Choroideremia. Genes 10:10, pages 738.
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Patricia A. Leake, Stephen J. Rebscher, Chantale Dore‘ & Omar Akil. (2019) AAV-Mediated Neurotrophin Gene Therapy Promotes Improved Survival of Cochlear Spiral Ganglion Neurons in Neonatally Deafened Cats: Comparison of AAV2-hBDNF and AAV5-hGDNF. Journal of the Association for Research in Otolaryngology 20:4, pages 341-361.
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Anne Louise Askou, Sidsel Alsing, Josephine N.E. Benckendorff, Andreas Holmgaard, Jacob Giehm Mikkelsen, Lars Aagaard, Toke Bek & Thomas J. Corydon. (2019) Suppression of Choroidal Neovascularization by AAV-Based Dual-Acting Antiangiogenic Gene Therapy. Molecular Therapy - Nucleic Acids 16, pages 38-50.
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Tuyen Ong, Mark E. Pennesi, David G. Birch, Byron L. Lam & Stephen H. Tsang. (2019) Adeno-Associated Viral Gene Therapy for Inherited Retinal Disease. Pharmaceutical Research 36:2.
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