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 …

Inherited and age-related retinal degeneration is the hallmark of a large group of
heterogeneous diseases and is the main cause of untreatable blindness today. Genetic …

We recently developed adeno-associated virus (AAV) capsids to facilitate efficient and
noninvasive gene transfer to the central and peripheral nervous systems. However, a …

Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR‐associated
enzyme (Cas) is a naturally occurring genome editing tool adopted from the prokaryotic …

Genetic intervention is increasingly being explored as a therapeutic option for debilitating
disorders of the central nervous system. The safety and efficacy of gene therapies rely upon …

X-linked retinopathies represent a significant proportion of monogenic retinal disease. They
include progressive and stationary conditions, with and without syndromic features. Many …

Retinal gene therapy has had unprecedented success in generating treatments that can halt
vision loss. However, immunogenic response and long-term toxicity with the use of viral …

Hereditary hearing loss often results from mutation of genes expressed by cochlear hair
cells. Gene addition using AAV vectors has shown some efficacy in mouse models, but …

CRISPR-Cas-based genome editing technologies could, in principle, be used to treat a wide
variety of inherited diseases, including genetic disorders of vision. Programmable CRISPR …

Gene therapy has entered a new era after decades-long efforts, where the recombinant
adeno-associated virus (AAV) has stood out as the most potent vector for in vivo gene …