Stargardt disease: clinical features, molecular genetics, animal models and therapeutic options

P Tanna, RW Strauss, K Fu**ami… - British Journal of …, 2017‏ - bjo.bmj.com
Stargardt disease (STGD1; MIM 248200) is the most prevalent inherited macular dystrophy
and is associated with disease-causing sequence variants in the gene ABCA4. Significant …

Inherited retinal diseases: Therapeutics, clinical trials and end points—A review

M Georgiou, K Fu**ami… - Clinical & Experimental …, 2021‏ - Wiley Online Library
Inherited retinal diseases (IRDs) are a clinically and genetically heterogeneous group of
disorders characterised by photoreceptor degeneration or dysfunction. These disorders …

Cell atlas of the human fovea and peripheral retina

W Yan, YR Peng, T van Zyl, A Regev, K Shekhar… - Scientific reports, 2020‏ - nature.com
Most irreversible blindness results from retinal disease. To advance our understanding of
the etiology of blinding diseases, we used single-cell RNA-sequencing (scRNA-seq) to …

Artificial intelligence in retinal screening using OCT images: A review of the last decade (2013–2023)

MH Akpinar, A Sengur, O Faust, L Tong… - Computer methods and …, 2024‏ - Elsevier
Background and objectives Optical coherence tomography (OCT) has ushered in a
transformative era in the domain of ophthalmology, offering non-invasive imaging with high …

Release of extracellular membrane particles carrying the stem cell marker prominin-1 (CD133) from neural progenitors and other epithelial cells

AM Marzesco, P Janich… - Journal of cell …, 2005‏ - journals.biologists.com
Apical plasma membrane constituents of mammalian neural stem/progenitor cells have
recently been implicated in maintaining their stem/progenitor cell state. Here, we report that …

The role of inflammation in the pathogenesis of age-related macular degeneration

LA Donoso, D Kim, A Frost, A Callahan… - Survey of …, 2006‏ - Elsevier
Age-related macular degeneration (AMD), the leading cause of blindness in the elderly, is a
complex disease to study because of the potential role of demographic, environmental, and …

[HTML][HTML] Clinical and molecular characteristics of childhood-onset Stargardt disease

K Fu**ami, J Zernant, RK Chana, GA Wright… - Ophthalmology, 2015‏ - Elsevier
Purpose To describe the clinical and molecular characteristics of patients with childhood-
onset Stargardt disease (STGD). Design Retrospective case series. Participants Forty-two …

Macular dystrophies: clinical and imaging features, molecular genetics and therapeutic options

N Rahman, M Georgiou, KN Khan… - British Journal of …, 2020‏ - bjo.bmj.com
Macular dystrophies (MDs) consist of a heterogeneous group of disorders that are
characterised by bilateral symmetrical central visual loss. Advances in genetic testing over …

The spectrum of retinal dystrophies caused by mutations in the peripherin/RDS gene

CJF Boon, AI den Hollander, CB Hoyng… - Progress in retinal and …, 2008‏ - Elsevier
Peripherin/rds is an integral membrane glycoprotein, mainly located in the rod and cone
outer segments. The relevance of this protein to photoreceptor outer segment morphology …

[HTML][HTML] A longitudinal study of Stargardt disease: quantitative assessment of fundus autofluorescence, progression, and genotype correlations

K Fu**ami, N Lois, R Mukherjee… - … & visual science, 2013‏ - jov.arvojournals.org
Purpose.: We characterized subtypes of fundus autofluorescence (AF) and the progression
of retinal atrophy, and correlated these findings with genotype in Stargardt disease …