[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 …

[HTML][HTML] An Overview of the Genetics of ABCA4 Retinopathies, an Evolving Story

S Al-Khuzaei, S Broadgate, CR Foster, M Shah, J Yu… - Genes, 2021‏ - mdpi.com
Stargardt disease (STGD1) and ABCA4 retinopathies (ABCA4R) are caused by pathogenic
variants in the ABCA4 gene inherited in an autosomal recessive manner. The gene encodes …

[HTML][HTML] ABCA4-associated disease as a model for missing heritability in autosomal recessive disorders: novel noncoding splice, cis-regulatory, structural, and …

M Bauwens, A Garanto, R Sangermano, S Naessens… - Genetics in …, 2019‏ - Elsevier
Purpose ABCA4-associated disease, a recessive retinal dystrophy, is hallmarked by a large
proportion of patients with only one pathogenic ABCA4 variant, suggestive for missing …

Genetic characteristics and epidemiology of inherited retinal degeneration in Taiwan

TC Chen, DS Huang, CW Lin, CH Yang… - NPJ genomic …, 2021‏ - nature.com
Inherited retinal degenerations (IRDs) are a group of phenotypically and genotypically
heterogeneous disorders with substantial socioeconomic impact. In this cohort study, we …

Cis-acting modifiers in the ABCA4 locus contribute to the penetrance of the major disease-causing variant in Stargardt disease

W Lee, J Zernant, T Nagasaki, LL Molday… - Human molecular …, 2021‏ - academic.oup.com
Over 1200 variants in the ABCA4 gene cause a wide variety of retinal disease phenotypes,
the best known of which is autosomal recessive Stargardt disease (STGD1). Disease …

Lessons learned from quantitative fundus autofluorescence

JR Sparrow, T Duncker, K Schuerch, M Paavo… - Progress in retinal and …, 2020‏ - Elsevier
Quantitative fundus autofluorescence (qAF) is an approach that is built on a confocal
scanning laser platform and used to measure the intensity of the inherent autofluorescence …

The common ABCA4 variant p. Asn1868Ile shows nonpenetrance and variable expression of Stargardt disease when present in trans with severe variants

EH Runhart, R Sangermano… - … & visual science, 2018‏ - iovs.arvojournals.org
Purpose: To assess the occurrence and the disease expression of the common p.
Asn1868Ile variant in patients with Stargardt disease (STGD1) harboring known, monoallelic …

Stargardt disease and progress in therapeutic strategies

D Huang, RC Heath Jeffery, MT Aung-Htut… - Ophthalmic …, 2022‏ - Taylor & Francis
Background Stargardt disease (STGD1) is an autosomal recessive retinal dystrophy due to
mutations in ABCA4, characterized by subretinal deposition of lipofuscin-like substances …

A genotype-phenotype correlation matrix for ABCA4 disease based on long-term prognostic outcomes

W Lee, J Zernant, PY Su, T Nagasaki, SH Tsang… - JCI …, 2022‏ - pmc.ncbi.nlm.nih.gov
Background More than 1500 variants in the ATP-binding cassette, sub-family A, member 4
(ABCA4), locus underlie a heterogeneous spectrum of retinal disorders ranging from …

Stargardt disease: Multimodal imaging: A review

RC Heath Jeffery, FK Chen - Clinical & Experimental …, 2021‏ - Wiley Online Library
Stargardt disease (STGD1) is an autosomal recessive retinal dystrophy, characterised by
bilateral progressive central vision loss and subretinal deposition of lipofuscin‐like …