Associated Papers
CRISPR as a therapeutic tool for inherited retinal degenerations: Advances, challenges, and future directions.
Galdikaite-Braziene E et al.
Molecular Aspects of Medicine, 2026
RNA Sequencing and Targeted Knockdown Reveal miR-142a-5p as a Driver of Retinal Degeneration in rd1 Mice.
Yang N et al.
Biology, 2026
Reactive Oxygen Species Regulation Across Retinitis Pigmentosa Animal Models: A 25-Year Systematized Review.
de Freitas Azevedo-Repossi R et al.
Molecular Neurobiology, 2025
The role of miRNAs in retinal physiology and in Inherited Retinal Disorders.
Brandi P et al.
Physiology (bethesda, Md.), 2025
Transcriptional downregulation of rhodopsin is associated with desensitization of rods to light-induced damage in a murine model of retinitis pigmentosa.
Takita S et al.
Human Molecular Genetics, 2025
Cyclic nucleotide signaling as a drug target in retinitis pigmentosa.
Vainionpää K et al.
Febs Letters, 2025
Genetic and bioinformatic analysis of RHO and PRPH2 variants in north Indian retinitis pigmentosa patients.
Bhardwaj A et al.
Japanese Journal of Ophthalmology, 2025
In vivo efficacy of NRL knockdown with cell-penetrating siRNA in retinal degeneration.
Lee H et al.
Scientific Reports, 2025
Mechanisms of photoreceptor protection upon targeting the Nrl-Nr2e3 pathway.
Murphy DP et al.
Proceedings of the National Academy of Sciences of the United States of America, 2025
Understanding TAK1 deficiency in microglia: Dual mechanisms for photoreceptor protection in a mouse model of retinitis pigmentosa.
Zhang J et al.
Proceedings of the National Academy of Sciences of the United States of America, 2025
Reactivating the phototransduction cascade with a mutation agnostic gene therapy preserves vision in rod-cone dystrophies.
Simon CJ et al.
Iscience, 2025
In vitro high-content screening reveals miR-429 as a protective molecule in photoreceptor degeneration.
Petrogiannakis G et al.
Molecular Therapy. Nucleic Acids, 2025
RHO Variants and Autosomal Dominant Retinitis Pigmentosa: Insights from the Italian Genetic Landscape.
Trastulli G et al.
Genes, 2024
Knockout and Replacement Gene Surgery to Treat Rhodopsin-Mediated Autosomal Dominant Retinitis Pigmentosa.
Sun X et al.
Human Gene Therapy, 2024
Molecular mechanisms underlying inherited photoreceptor degeneration as targets for therapeutic intervention.
Bighinati A et al.
Frontiers in Cellular Neuroscience, 2024
Genetic treatment for autosomal dominant inherited retinal dystrophies: approaches, challenges and targeted genotypes.
Daich Varela M et al.
The British Journal of Ophthalmology, 2023
Deciphering the role of cytoplasmic domain of Channelrhodopsin in modulation of the interactome and SUMOylome of Chlamydomonas reinhardtii.
Sharma K et al.
International Journal of Biological Macromolecules, 2023
Allele-specific gene-editing approach for vision loss restoration in RHO-associated retinitis pigmentosa.
Liu X et al.
Elife, 2023
Retinitis Pigmentosa: Novel Therapeutic Targets and Drug Development.
Wu KY et al.
Pharmaceutics, 2023
Cost-effective sequence analysis of 113 genes in 1,192 probands with retinitis pigmentosa and Leber congenital amaurosis.
Panneman DM et al.
Frontiers in Cell and Developmental Biology, 2023
Rhodopsin-associated retinal dystrophy: Disease mechanisms and therapeutic strategies.
Zhen F et al.
Frontiers in Neuroscience, 2023
Disease-causing mutations in genes encoding transcription factors critical for photoreceptor development.
Sun C et al.
Frontiers in Molecular Neuroscience, 2023
miR-181a/b downregulation: a mutation-independent therapeutic approach for inherited retinal diseases.
Carrella S et al.
Embo Molecular Medicine, 2022
Gene Therapy for Rhodopsin Mutations.
Lewin AS et al.
Cold Spring Harbor Perspectives in Medicine, 2022
Molecular basis for variations in the sensitivity of pathogenic rhodopsin variants to 9-cis-retinal.
Roushar FJ et al.
The Journal of Biological Chemistry, 2022
CRISPR DNA Base Editing Strategies for Treating Retinitis Pigmentosa Caused by Mutations in Rhodopsin.
Kaukonen M et al.
Genes, 2022
Long-term vitamin A supplementation in a preclinical mouse model for RhoD190N-associated retinitis pigmentosa.
Cui X et al.
Human Molecular Genetics, 2022
The role of motor proteins in photoreceptor protein transport and visual function.
Radhakrishnan R et al.
Ophthalmic Genetics, 2022
MicroRNA regulation of critical retinal pigment epithelial functions.
Du SW et al.
Trends in Neurosciences, 2022
Genetics of Inherited Retinal Diseases in Understudied Populations.
Kannabiran C et al.
Frontiers in Genetics, 2022
Induction of Autophagy Promotes Clearance of RHOP23H Aggregates and Protects From Retinal Degeneration.
Intartaglia D et al.
Frontiers in Aging Neuroscience, 2022
Identification of a novel RHO heterozygous nonsense mutation in a Chinese family with autosomal dominant retinitis pigmentosa.
Liu W et al.
Bmc Ophthalmology, 2021
Gene Therapy for Rhodopsin-associated Autosomal Dominant Retinitis Pigmentosa.
Massengill MT et al.
International Ophthalmology Clinics, 2021
Mirtron-mediated RNA knockdown/replacement therapy for the treatment of dominant retinitis pigmentosa.
Orlans HO et al.
Nature Communications, 2021
Allele-specific editing ameliorates dominant retinitis pigmentosa in a transgenic mouse model.
Patrizi C et al.
American Journal of Human Genetics, 2021
Pre-mRNA Processing Factors and Retinitis Pigmentosa: RNA Splicing and Beyond.
Yang C et al.
Frontiers in Cell and Developmental Biology, 2021
Toward the Treatment of Inherited Diseases of the Retina Using CRISPR-Based Gene Editing.
Hernández-Juárez J et al.
Frontiers in Medicine, 2021
Structure network-based landscape of rhodopsin misfolding by mutations and algorithmic prediction of small chaperone action.
Felline A et al.
Computational and Structural Biotechnology Journal, 2021