[HTML][HTML] Progress in treating inherited retinal diseases: early subretinal gene therapy clinical trials and candidates for future initiatives
AV Garafalo, AV Cideciyan, E Héon, R Sheplock… - Progress in retinal and …, 2020 - Elsevier
Due to improved phenotyping and genetic characterization, the field of 'incurable'and
'blinding'inherited retinal diseases (IRDs) has moved substantially forward. Decades of …
'blinding'inherited retinal diseases (IRDs) has moved substantially forward. Decades of …
Fundus autofluorescence imaging
Fundus autofluorescence (FAF) imaging is an in vivo imaging method that allows for
topographic mapping of naturally or pathologically occurring intrinsic fluorophores of the …
topographic mapping of naturally or pathologically occurring intrinsic fluorophores of the …
Phenotyping and genotyping inherited retinal diseases: Molecular genetics, clinical and imaging features, and therapeutics of macular dystrophies, cone and cone-rod …
Inherited retinal diseases (IRD) are a leading cause of blindness in the working age
population and children. The scope of this review is to familiarise clinicians and scientists …
population and children. The scope of this review is to familiarise clinicians and scientists …
Structure and function of ABCA4 and its role in the visual cycle and Stargardt macular degeneration
RS Molday, FA Garces, JF Scortecci… - Progress in retinal and eye …, 2022 - Elsevier
ABCA4 is a member of the superfamily of ATP-binding cassette (ABC) transporters that is
preferentially localized along the rim region of rod and cone photoreceptor outer segment …
preferentially localized along the rim region of rod and cone photoreceptor outer segment …
[HTML][HTML] Endpoints for clinical trials in ophthalmology
With the identification of novel targets, the number of interventional clinical trials in
ophthalmology has increased. Visual acuity has for a long time been considered the gold …
ophthalmology has increased. Visual acuity has for a long time been considered the gold …
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 …
characterised by bilateral symmetrical central visual loss. Advances in genetic testing over …
A phase I clinical trial of human embryonic stem cell‐derived retinal pigment epithelial cells for early‐stage Stargardt macular degeneration: 5‐years' follow‐up
SY Li, Y Liu, L Wang, F Wang, TT Zhao, QY Li… - Cell …, 2021 - Wiley Online Library
Objectives To evaluate the long‐term biosafety and efficacy of transplantation of human
embryonic stem cells‐derived retinal pigment epithelial (hESC‐RPE) cells in early‐stage of …
embryonic stem cells‐derived retinal pigment epithelial (hESC‐RPE) cells in early‐stage of …
[HTML][HTML] Advancing clinical trials for inherited retinal diseases: recommendations from the Second Monaciano Symposium
DA Thompson, A Iannaccone, RR Ali… - … vision science & …, 2020 - arvojournals.org
Major advances in the study of inherited retinal diseases (IRDs) have placed efforts to
develop treatments for these blinding conditions at the forefront of the emerging field of …
develop treatments for these blinding conditions at the forefront of the emerging field of …
[HTML][HTML] Fundus autofluorescence and clinical applications
C Pole, H Ameri - Journal of Ophthalmic & Vision Research, 2021 - ncbi.nlm.nih.gov
Fundus autofluorescence (FAF) has allowed in vivo mapping of retinal metabolic
derangements and structural changes not possible with conventional color imaging. Incident …
derangements and structural changes not possible with conventional color imaging. Incident …
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 …
bilateral progressive central vision loss and subretinal deposition of lipofuscin‐like …