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Gene therapies in clinical development to treat retinal disorders
ME McClements, MEAA Elsayed, L Major… - Molecular Diagnosis & …, 2024 - Springer
Gene therapies have emerged as promising treatments in clinical development for various
retinal disorders, offering hope to patients with inherited degenerative eye conditions …
retinal disorders, offering hope to patients with inherited degenerative eye conditions …
[HTML][HTML] AAV-RPGR gene therapy rescues Opsin mislocalisation in a human retinal organoid model of RPGR-associated X-linked retinitis pigmentosa
PE Sladen, A Naeem, T Adefila-Ideozu… - International Journal of …, 2024 - mdpi.com
Variants within the Retinitis Pigmentosa GTPase regulator (RPGR) gene are the
predominant cause of X-Linked Retinitis Pigmentosa (XLRP), a common and severe form of …
predominant cause of X-Linked Retinitis Pigmentosa (XLRP), a common and severe form of …
X-linked retinitis pigmentosa gene therapy: preclinical aspects
V Mansouri - Ophthalmology and Therapy, 2023 - Springer
The most common inherited eye disease is retinitis pigmentosa (RP). X-linked RP (XLRP) is
one of the most severe types of RP, with a considerable disease burden. Patients with XLRP …
one of the most severe types of RP, with a considerable disease burden. Patients with XLRP …
[HTML][HTML] Overcoming the challenges to clinical development of X-linked retinitis pigmentosa therapies: proceedings of an expert panel
DG Birch, JK Cheetham, SP Daiger… - … Vision Science & …, 2023 - jov.arvojournals.org
X-linked retinitis pigmentosa (XLRP) is a rare inherited retinal disease manifesting as
impaired night vision and peripheral vision loss that progresses to legal blindness. Although …
impaired night vision and peripheral vision loss that progresses to legal blindness. Although …
[HTML][HTML] Pre-Clinical and Clinical Advances in Gene Therapy of X-Linked Retinitis Pigmentosa: Hope on the Horizon
NA Pechnikova, M Poimenidou, I Iliadis… - Journal of Clinical …, 2025 - mdpi.com
X-linked retinitis pigmentosa (XLRP) is a severe inherited retinal degenerative disease
characterized by progressive loss of photoreceptors and retinal pigment epithelium, leading …
characterized by progressive loss of photoreceptors and retinal pigment epithelium, leading …
Challenges in Value Assessment for One-Time Gene Therapies for Inherited Retinal Diseases: Are We Turning a Blind Eye?
J Hitch, T Denee, S Brassel, J Lee, M Michaelides… - Value in Health, 2025 - Elsevier
Objectives X-linked retinitis pigmentosa (XLRP) is a rare inherited retinal disease with no
available treatment. Gene therapies in clinical trials will pose challenges for health …
available treatment. Gene therapies in clinical trials will pose challenges for health …
RWC Update: Enhanced ILM Peeling; Retinal Gene Therapy; Laser-Induced Retinal Break and Vitreous Hemorrhage
A 60-year-old woman underwent vitrectomy and internal limiting membrane (ILM) peeling for
a primary macular hole. To enhance visualization, we applied Brilliant Blue dye meticulously …
a primary macular hole. To enhance visualization, we applied Brilliant Blue dye meticulously …
[PDF][PDF] Promising gene therapies for ocular conditions
A KHAMISE - eyenews.uk.com
ADVM-022, developed by Adverum Biotechnologies, is an innovative gene therapy aimed at
treating wet age-related macular degeneration (AMD). This therapy employs a novel …
treating wet age-related macular degeneration (AMD). This therapy employs a novel …
[HTML][HTML] My Top Five: Promising gene therapies for ocular conditions
A Khamise - eyenews.uk.com
Gene therapy, a pivotal advancement in modern medicine, particularly shines in
ophthalmology. By targeting defective genes with engineered vectors, this approach …
ophthalmology. By targeting defective genes with engineered vectors, this approach …