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 …

Stem cell transplantation holds great promise as a potential treatment for currently incurable
retinal degenerative diseases that cause poor vision and blindness. Recently, safety data …

The mammalian retina displays no intrinsic regenerative capacities, therefore retinal
degenerative diseases such as age-related macular degeneration (AMD) or retinitis …

Retinitis pigmentosa (RP) comprises a group of inherited retinal dystrophies characterized
by the degeneration of rod photoreceptors, followed by the degeneration of cone …

Inherited retinal diseases (IRDs) comprise a heterogeneous group of genetic disorders
affecting the retina. Caused by mutations in over 300 genes, IRDs result in visual impairment …

The human retina is a complex tissue responsible for detecting photons of light and
converting information from these photons into the neurochemical signals interpreted as …

Bilallelic variants in the USH2A gene can cause Usher syndrome type 2 and non-syndromic
retinitis pigmentosa. In both disorders, the retinal phenotype involves progressive rod …

Inherited retinal diseases (IRDs) are a heterogenous group of orphan eye diseases that
typically result from monogenic mutations and are considered attractive targets for gene …

The retina has been at the forefront of translational gene therapy. Proof-of-concept that gene
therapy could restore vision in a large animal led to the initiation of the first successful …

Challenges to the widespread application of gene therapy with adeno-associated viral
(AAV) vectors include dominant conditions due to gain-of-function mutations which require …