A cross-sectional study to assess the clinical utility of modern visual function assessments in patients with inherited retinal disease: a mixed methods observational study protocol.
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Taylor LJ. et al, (2023), BMC Ophthalmol, 23
A Review of CRISPR Tools for Treating Usher Syndrome: Applicability, Safety, Efficiency, and In Vivo Delivery.
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Major L. et al, (2023), Int J Mol Sci, 24
AAV2-Mediated Gene Therapy for Choroideremia: 5-Year Results and Alternate Anti-sense Oligonucleotide Therapy
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Zhai Y. et al, (2023), American Journal of Ophthalmology, 248, 145 - 156
Erratum: Enhancement of Adeno-Associated Virus-Mediated Gene Therapy Using Hydroxychloroquine in Murine and Human Tissues.
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Chandler LC. et al, (2023), Mol Ther Methods Clin Dev, 28
Changes in Retinal Sensitivity Associated With Cotoretigene Toliparvovec in X-Linked Retinitis Pigmentosa With RPGR Gene Variations
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von Krusenstiern L. et al, (2023), JAMA Ophthalmology, 141, 275 - 275
Assessment of Scotopic Function in Rod-Cone Inherited Retinal Degeneration With the Scotopic Macular Integrity Assessment.
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Jolly JK. et al, (2023), Transl Vis Sci Technol, 12
Future Perspectives of Prime Editing for the Treatment of Inherited Retinal Diseases.
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Hansen S. et al, (2023), Cells, 12
Is RPGR-related retinal dystrophy associated with systemic disease? A case series
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Han RC. et al, (2023), Ophthalmic Genetics, 1 - 8
Harnessing the potential of practice-based clinical optometry research in the United Kingdom.
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Taylor LJ. et al, (2022), Ophthalmic Physiol Opt
Impaired glutamylation of RPGR ORF15 underlies the cone-dominated phenotype associated with truncating distal ORF15 variants
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Cehajic-Kapetanovic J. et al, (2022), Proceedings of the National Academy of Sciences, 119
Compound dominant-null heterozygosity in a family with RP1-related retinal dystrophy
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Buckley TMW. et al, (2022), American Journal of Ophthalmology Case Reports, 28
Response: 'letter to the editor: emerging gene therapy products for RPGR-associated X-linked retinitis pigmentosa'.
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Martinez-Fernandez de la Camara C. et al, (2022), Expert Opin Emerg Drugs, 27, 449 - 450
Potential CRISPR Base Editing Therapeutic Options in a Sorsby Fundus Dystrophy Patient.
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Elsayed MEAA. et al, (2022), Genes (Basel), 13
Emerging gene therapy products for RPGR-associated X-linked retinitis pigmentosa
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Martinez-Fernandez de la Camara C. et al, (2022), Expert Opinion on Emerging Drugs, 27, 431 - 443
Minicircle Delivery to the Neural Retina as a Gene Therapy Approach.
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Staurenghi F. et al, (2022), Int J Mol Sci, 23
New CRISPR Tools to Correct Pathogenic Mutations in Usher Syndrome.
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Major L. et al, (2022), Int J Mol Sci, 23
Gene therapy for inherited retinal disease: long-term durability of effect.
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Leroy BP. et al, (2022), Ophthalmic Res
Non-Viral Delivery of CRISPR/Cas Cargo to the Retina Using Nanoparticles: Current Possibilities, Challenges, and Limitations.
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Salman A. et al, (2022), Pharmaceutics, 14
MERTK missense variants in three patients with retinitis pigmentosa.
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Poli FE. et al, (2022), Ophthalmic Genet, 1 - 9
Phenotypic and Genetic Characteristics in a Cohort of Patients with Usher Genes
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Feenstra HM. et al, (2022), Genes, 13, 1423 - 1423