[HTML][HTML] Clinical spectrum, genetic complexity and therapeutic approaches for retinal disease caused by ABCA4 mutations

FPM Cremers, W Lee, RWJ Collin… - Progress in retinal and eye …, 2020‏ - Elsevier
The ABCA4 protein (then called a “rim protein”) was first identified in 1978 in the rims and
incisures of rod photoreceptors. The corresponding gene, ABCA4, was cloned in 1997, and …

Inherited retinal diseases: linking genes, disease-causing variants, and relevant therapeutic modalities

N Schneider, Y Sundaresan, P Gopalakrishnan… - Progress in retinal and …, 2022‏ - Elsevier
Inherited retinal diseases (IRDs) are a clinically complex and heterogenous group of visual
impairment phenotypes caused by pathogenic variants in at least 277 nuclear and …

[HTML][HTML] De novo birth of functional microproteins in the human lineage

N Vakirlis, Z Vance, KM Duggan, A McLysaght - Cell reports, 2022‏ - cell.com
Small open reading frames (sORFs) can encode functional" microproteins" that perform
crucial biological tasks. However, their size makes them less amenable to genomic analysis …

Genetic epidemiology of inherited retinal diseases in a large patient cohort followed at a single center in Italy

M Karali, F Testa, V Di Iorio, A Torella, R Zeuli… - Scientific Reports, 2022‏ - nature.com
Inherited retinal diseases (IRDs) are the leading cause of vision loss in the working-age
population. We performed a retrospective epidemiological study to determine the genetic …

Genetic architecture of inherited retinal degeneration in Germany: A large cohort study from a single diagnostic center over a 9‐year period

N Weisschuh, CD Obermaier, F Battke… - Human …, 2020‏ - Wiley Online Library
We aimed to unravel the molecular genetic basis of inherited retinal degeneration (IRD) in a
comprehensive cohort of patients diagnosed in the largest center for IRD in Germany. A …

Visual function restoration in a mouse model of Leber congenital amaurosis via therapeutic base editing

DH Jo, HK Jang, CS Cho, JH Han, G Ryu… - … Therapy Nucleic Acids, 2023‏ - cell.com
Leber congenital amaurosis (LCA), an inherited retinal degeneration, causes severe visual
dysfunction in children and adolescents. In patients with LCA, pathogenic variants, such as …

Copy-number variation contributes 9% of pathogenicity in the inherited retinal degenerations

E Zampaglione, B Kinde, EM Place… - Genetics in …, 2020‏ - nature.com
Purpose Current sequencing strategies can genetically solve 55–60% of inherited retinal
degeneration (IRD) cases, despite recent progress in sequencing. This can partially be …

Updating the genetic landscape of inherited retinal dystrophies

B García Bohórquez, E Aller… - Frontiers in cell and …, 2021‏ - frontiersin.org
Inherited retinal dystrophies (IRD) are a group of diseases characterized by the loss or
dysfunction of photoreceptors and a high genetic and clinical heterogeneity. Currently, over …

[HTML][HTML] Inherited Retinal Diseases Due to RPE65 Variants: From Genetic Diagnostic Management to Therapy

M Aoun, I Passerini, P Chiurazzi, M Karali… - International journal of …, 2021‏ - mdpi.com
Inherited retinal diseases (IRDs) are a heterogeneous group of conditions that include
retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA) and early-onset severe …

[HTML][HTML] Mutation spectrum of PRPF31, genotype-phenotype correlation in retinitis pigmentosa, and opportunities for therapy

G Wheway, A Douglas, D Baralle, E Guillot - Experimental eye research, 2020‏ - Elsevier
Pathogenic variants in pre-messenger RNA (pre-mRNA) splicing factor 31, PRPF31, are the
second most common genetic cause of autosomal dominant retinitis pigmentosa (adRP) in …