Many genetic diseases and undesirable traits are due to base-pair alterations in genomic
DNA. Base-editing, the newest evolution of clustered regularly interspaced short palindromic …

Until recently, there was no approved treatment for a retinal degenerative disease.
Subretinal injection of a recombinant adeno-associated virus (AAV) delivering the normal …

Leber congenital amaurosis type 10 is a severe retinal dystrophy caused by mutations in the
CEP290 gene,. We developed EDIT-101, a candidate genome-editing therapeutic, to …

One of the major questions in human genetics is what percentage of individuals in the
general population carry a disease-causing mutation. Based on publicly available …

Purpose In a large cohort of molecularly characterized inherited retinal disease (IRD)
families, we investigated proportions with disease attributable to causative variants in each …

A resurgence of interest and investment in the field of gene therapy, driven in large part by
advances in viral vector technology, has recently culminated in United States Food and Drug …

Due to improved phenotyping and genetic characterization, the field of 'incurable'and
'blinding'inherited retinal diseases (IRDs) has moved substantially forward. Decades of …

Cell replacement therapy is a promising treatment for irreversible retinal cell death in diverse
diseases, such as age-related macular degeneration (AMD), Stargardt's disease, retinitis …

Macular and cone/cone-rod dystrophies (MD/CCRD) demonstrate a broad genetic and
phenotypic heterogeneity, with retinal alterations solely or predominantly involving the …

Inherited retinal diseases (IRDs) are the leading cause of vision loss in the working-age
population. We performed a retrospective epidemiological study to determine the genetic …