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Human gene therapy, ISSN 1043-0342, 02/2016, Volume 27, Issue 2, pp. 134 - 147
... such as retinitis pigmentosa, Stargardt disease, and Leber congenital amaurosis, as well as in complex age-related diseases... 
Reviews | PHOTORECEPTOR PRECURSORS | MEDICINE, RESEARCH & EXPERIMENTAL | RESTORES VISUAL RESPONSES | RETINAL PIGMENTED EPITHELIUM | PLURIPOTENT STEM-CELLS | CONE VIABILITY FACTOR | LEBER CONGENITAL AMAUROSIS | PHASE-I TRIAL | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | OPTIMIZED ALLOTOPIC EXPRESSION | MOUSE MODEL | GENETICS & HEREDITY | RETINITIS-PIGMENTOSA | Leber Congenital Amaurosis - pathology | Cell- and Tissue-Based Therapy - methods | Dependovirus - genetics | Retina - metabolism | Optogenetics - methods | Blindness - metabolism | Humans | Blindness - pathology | Leber Congenital Amaurosis - metabolism | Lentivirus - genetics | cis-trans-Isomerases - metabolism | Blindness - therapy | Leber Congenital Amaurosis - therapy | Genetic Vectors - chemistry | Genetic Vectors - metabolism | Retinitis Pigmentosa - genetics | Retinitis Pigmentosa - metabolism | Clinical Trials as Topic | Leber Congenital Amaurosis - genetics | Blindness - genetics | Macular Degeneration - metabolism | Macular Degeneration - congenital | cis-trans-Isomerases - genetics | Macular Degeneration - genetics | Macular Degeneration - therapy | Retinitis Pigmentosa - therapy | CRISPR-Cas Systems | Adenoviruses, Human - genetics | Retina - pathology | Retinitis Pigmentosa - pathology | Genetic Therapy - methods | Macular Degeneration - pathology | Macular degeneration | Therapy | Cell death | Retinal cells | Retinitis pigmentosa | Blindness | Disorders | Retinal degeneration | Retina | Retinitis | Age | Life Sciences
Journal Article
British Journal of Ophthalmology, ISSN 0007-1161, 09/2017, Volume 101, Issue 9, pp. 1147 - 1154
Journal Article
RETINA-THE JOURNAL OF RETINAL AND VITREOUS DISEASES, ISSN 0275-004X, 11/2019, Volume 39, Issue 11, pp. 2059 - 2069
.... It is estimated to affect approximately 1 in 50,000 male patients. It is characterized by progressive degeneration of the retinal pigment epithelium, choroid, and photoreceptors, resulting in visual impairment and blindness... 
MACULAR DEGENERATION | RPE65 MUTATIONS | NATURAL-HISTORY | PIGMENT-EPITHELIUM | TRANSPLANTATION | LEBER CONGENITAL AMAUROSIS | SUBRETINAL INJECTION | choroideremia | VISUAL FUNCTION | OPHTHALMOLOGY | GENE-THERAPY | QUALITY-OF-LIFE | inherited retinal degeneration
Journal Article