Gene therapy in the early stages of retinal degeneration
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Expanding the genotypic and phenotypic spectra with a novel variant in the ciliopathy gene, CFAP410, associated with selective cone degeneration.
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Borchert GA. et al, (2024), Ophthalmic Genet, 1 - 7
Genetic therapies and potential therapeutic applications of CRISPR activators in the eye.
Journal article
Ng BW. et al, (2024), Prog Retin Eye Res
Investigating the impact of asymmetric macular sensitivity on visual acuity chart reading in choroideraemia.
Journal article
Baffour-Awuah KA. et al, (2024), Ophthalmic Physiol Opt
An Open-Label Phase II Study Assessing the Safety of Bilateral, Sequential Administration of Retinal Gene Therapy in Participants with Choroideremia: The GEMINI Study.
Journal article
MacLaren RE. et al, (2024), Hum Gene Ther
Gene Therapies in Clinical Development to Treat Retinal Disorders.
Journal article
McClements ME. et al, (2024), Mol Diagn Ther
Retinal characteristics of female choroideremia carriers: Multimodal imaging, microperimetry and genetics.
Journal article
Gocuk SA. et al, (2024), Ophthalmol Retina
Robot-Assisted Eye Surgery: A Systematic Review of Effectiveness, Safety, and Practicality in Clinical Settings.
Journal article
Thirunavukarasu AJ. et al, (2024), Transl Vis Sci Technol, 13
In Silico CRISPR-Cas-Mediated Base Editing Strategies for Early-Onset, Severe Cone-Rod Retinal Degeneration in Three Crumbs homolog 1 Patients, including the Novel Variant c.2833G>A.
Journal article
Shamsnajafabadi H. et al, (2024), Genes (Basel), 15
Rescue of cone and rod photoreceptor function in a CDHR1-model of age-related retinal degeneration.
Journal article
Yusuf IH. et al, (2024), Mol Ther
Assessment of Visual Function with Cotoretigene Toliparvovec in X-Linked Retinitis Pigmentosa in the Randomized XIRIUS Phase 2/3 Study.
Journal article
Lam BL. et al, (2024), Ophthalmology
A Prospective, Observational, Noninterventional Clinical Study of Participants With Choroideremia: The NIGHT Study.
Journal article
Maclaren RE. et al, (2024), Am J Ophthalmol, 263, 35 - 49
Optical Coherence Tomography Feature of Retinoschisis in CRB1-Associated Maculopathy.
Journal article
Bellingrath J-S. et al, (2024), JAMA Ophthalmol, 142, 158 - 161
CRISPR Manipulation of Age-Related Macular Degeneration Haplotypes in the Complement System: Potential Future Therapeutic Applications/Avenues.
Journal article
Salman A. et al, (2024), Int J Mol Sci, 25
A hypomorphic variant of choroideremia is associated with a novel intronic mutation that leads to exon skipping.
Journal article
Waldock WJ. et al, (2024), Ophthalmic Genet, 1 - 8
Age-related macular degeneration: suitability of optogenetic therapy for geographic atrophy.
Journal article
Borchert GA. et al, (2024), Front Neurosci, 18
Retinal Focal Nodular Gliosis (Vasoproliferative Tumors) Have Varied Clinical Courses Requiring Tailored Management: A Case Series
Journal article
Ie A. et al, (2024), Ocular Oncology and Pathology
Comment on Di Giosaffatte et al. A Novel Hypothesis on Choroideremia-Manifesting Female Carriers: Could CHM In-Frame Variants Exert a Dominant Negative Effect? A Case Report. Genes 2022, 13, 1268
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Fry LE. and MacLaren RE., (2023), Genes, 14