Biology and therapy of inherited retinal degenerative disease: insights from mouse models

S Veleri, CH Lazar, B Chang… - Disease models & …, 2015 - journals.biologists.com
Retinal neurodegeneration associated with the dysfunction or death of photoreceptors is a
major cause of incurable vision loss. Tremendous progress has been made over the last two …

A comprehensive review of retinal gene therapy

SE Boye, SL Boye, AS Lewin, WW Hauswirth - Molecular therapy, 2013 - cell.com
Blindness, although not life threatening, is a debilitating disorder for which few, if any
treatments exist. Ocular gene therapies have the potential to profoundly improve the quality …

Restoration of vision after de novo genesis of rod photoreceptors in mammalian retinas

K Yao, S Qiu, YV Wang, SJH Park, EJ Mohns, B Mehta… - Nature, 2018 - nature.com
In zebrafish, Müller glia (MG) are a source of retinal stem cells that can replenish damaged
retinal neurons and restore vision. In mammals, however, MG do not spontaneously re-enter …

AAV cis-regulatory sequences are correlated with ocular toxicity

W Xiong, DM Wu, Y Xue, SK Wang… - Proceedings of the …, 2019 - National Acad Sciences
Adeno-associated viral vectors (AAVs) have become popular for gene therapy, given their
many advantages, including their reduced inflammatory profile compared with that of other …

[HTML][HTML] Targeting necroptosis: a novel therapeutic option for retinal degenerative diseases

Q Zhang, X Hu, W Zhao, X Ban, Y Li… - … Journal of Biological …, 2023 - ncbi.nlm.nih.gov
The discovery of the necroptosis, a form of regulated necrosis that is mediated by receptor-
interacting protein kinase 1 (RIPK1), RIPK3, and mixed-lineage kinase domain-like …

Rod photoreceptors drive circadian photoentrainment across a wide range of light intensities

CM Altimus, AD Güler, NM Alam, AC Arman… - Nature …, 2010 - nature.com
In mammals, synchronization of the circadian pacemaker in the hypothalamus is achieved
through direct input from the eyes conveyed by intrinsically photosensitive retinal ganglion …

Photoresponse diversity among the five types of intrinsically photosensitive retinal ganglion cells

X Zhao, BK Stafford, AL Godin, WM King… - The Journal of …, 2014 - Wiley Online Library
Key points Intrinsically photosensitive retinal ganglion cells (ipRGCs) are a rare population
of retinal output neurons that drives subconscious physiological responses to light, eg …

Lighting a candle in the dark: advances in genetics and gene therapy of recessive retinal dystrophies

AI den Hollander, A Black, J Bennett… - The Journal of …, 2010 - Am Soc Clin Investig
Nonsyndromic recessive retinal dystrophies cause severe visual impairment due to the
death of photoreceptor and retinal pigment epithelium cells. These diseases until recently …

Achromatopsia: genetics and gene therapy

S Michalakis, M Gerhardt, G Rudolph… - Molecular diagnosis & …, 2022 - Springer
Achromatopsia (ACHM), also known as rod monochromatism or total color blindness, is an
autosomal recessively inherited retinal disorder that affects the cones of the retina, the type …

Two-photon imaging of nonlinear glutamate release dynamics at bipolar cell synapses in the mouse retina

BG Borghuis, JS Marvin, LL Looger… - Journal of …, 2013 - Soc Neuroscience
Alpha/Y-type retinal ganglion cells encode visual information with a receptive field
composed of nonlinear subunits. This nonlinear subunit structure enhances sensitivity to …