Researchers at the University of Oxford are using CRISPR-Cas Systems to correct mutations in an inherited retinal degenerative disease, which leads to blindness. Their work is published in the journal, Human Gene Therapy.
"Currently, the field is undergoing rapid development with a number of competing gene editing strategies, including allele-specific knock-down, base editing, prime editing, and RNA editing, are under investigation. Each offers a different balance of on-target editing efficiency versus off-target risks," said Kanmin Xue, of the University of Oxford. "Testing these newly-developed CRISPR technologies in human retinal tissue, organoids and in vivo will help to highlight the most-viable therapeutic approaches for treating inherited retinal diseases in the future."
Additionally, the researchers address epigenetic editing, the risks of retinal gene editing, and approaches in development to control Cas9 activity and optimize safety.
"The eye is an ideal target for in vivo gene editing. Dr. Xue's review provides an excellent overview of the current state of the art," says Editor-in-Chief of Human Gene Therapy Terence R. Flotte, MD.