Search results (39)
« Back to PublicationsSingle-cell and spatial transcriptomic analyses of gene therapy-associated retinal inflammation in non-human primates
Preprint
Sourd C. et al, (2025)
Gene Therapies in Clinical Development to Treat Retinal Disorders
Journal article
McClements ME. et al, (2024), Molecular Diagnosis & Therapy, 28, 575 - 591
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 Genetics, 45, 210 - 217
Gene Therapy Trial on X-Linked Retinitis Pigmentosa Caused by Mutations in RPGR
Chapter
de la Camara CM-F. et al, (2024), 271 - 283
Is RPGR-related retinal dystrophy associated with systemic disease? A case series
Journal article
Han RC. et al, (2023), Ophthalmic Genetics, 44, 577 - 584
Outcomes and Adverse Effects of Voretigene Neparvovec Treatment for Biallelic RPE65-Mediated Inherited Retinal Dystrophies in a Cohort of Patients from a Single Center
Journal article
Kiraly P. et al, (2023), Biomolecules, 13, 1484 - 1484
Developmental Expression of the Cell Cycle Regulator p16INK4a in Retinal Glial Cells: A Novel Marker for Immature Ocular Astrocytes?
Journal article
Martinez-Fernandez de la Camara C. et al, (2023), Journal of Histochemistry & Cytochemistry, 71, 301 - 320
Changes in Retinal Sensitivity Associated With Cotoretigene Toliparvovec in X-Linked Retinitis Pigmentosa With RPGR Gene Variations
Journal article
von Krusenstiern L. et al, (2023), JAMA Ophthalmology, 141, 275 - 275
Impaired glutamylation of RPGR ORF15 underlies the cone-dominated phenotype associated with truncating distal ORF15 variants
Journal article
Cehajic-Kapetanovic J. et al, (2022), Proceedings of the National Academy of Sciences, 119
Emerging gene therapy products for RPGR-associated X-linked retinitis pigmentosa
Journal article
Martinez-Fernandez de la Camara C. et al, (2022), Expert Opinion on Emerging Drugs, 27, 431 - 443
Characterizing the cellular immune response to subretinal AAV gene therapy in the murine retina
Journal article
Chandler LC. et al, (2021), Molecular Therapy - Methods & Clinical Development, 22, 52 - 65
Mirtron-mediated RNA knockdown/replacement therapy for the treatment of dominant retinitis pigmentosa
Journal article
Orlans HO. et al, (2021), Nature Communications, 12
Accurate Quantification of AAV Vector Genomes by Quantitative PCR
Journal article
Martinez-Fernandez de la Camara C. et al, (2021), Genes, 12, 601 - 601
Inclusion of PF68 Surfactant Improves Stability of rAAV Titer when Passed through a Surgical Device Used in Retinal Gene Therapy
Journal article
Patrício MI. et al, (2020), Molecular Therapy - Methods & Clinical Development, 17, 99 - 106
CRISPR Interference–Potential Application in Retinal Disease
Journal article
Peddle CF. et al, (2020), International Journal of Molecular Sciences, 21, 2329 - 2329