Year: 2017
Type: erratum
Abstract: The past decade has seen major advances in gene-based therapies, many of which show promise for translation to human disease. At the forefront of research in this field is ocular disease, as the eye lends itself to gene-based interventions due to its accessibility, relatively immune-privileged status, and ability to be non-invasively monitored. A landmark study in 2001 demonstrating successful gene therapy in a large-animal model for Leber congenital amaurosis set the stage for translation of these strategies from the bench to the bedside. Multiple clinical trials have since initiated for various retinal diseases, and further improvements in gene therapy techniques have engendered optimism for alleviating inherited blinding disorders. This article provides an overview of gene-based strategies for retinal disease, current clinical trials that engage these strategies, and the latest techniques in genome engineering, which could serve as the next frontline of therapeutic interventions.
Source: Genes
DOI: 10.3390/genes8020053
Language: English
Subfield: Molecular Biology
Domain: Life Sciences
Open Access status: gold
Estimated APC paid: $3,137
Awards: Core Facilities for Vision Research, G3423198228, Evaluating GWAS AMD Candidate Loci by Gene Editing in Human iPS Cells, Mechanistic Studies on Regenerative Medicine Approaches to Childhood Blindness, G8415332196, Defining Barriers to Gene Therapy, Gene Editing and Silencing in Phototransduction, Cancer Center Support Grant
OpenAlex ID: W2579979462