Gene editing techniques helpful in retinal degeneration treatment

A new study has found that gene-editing techniques like CRISPR-Cas hold the power to rectify inherited retinal degenerative mutations, which are the primary cause of blindness.

A new study has found that gene-editing techniques like CRISPR-Cas hold the power to rectify inherited retinal degenerative mutations, which are the primary cause of blindness. Published in the journal Human Gene Therapy, technological advances in gene editing, continuing safety concerns, and strategies to overcome these challenges are highlighted in the peer-reviewed journal Human Gene Therapy.

Kanmin Xue, University of Oxford, and co-authors state, "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," They added, "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."

Characterizing the rapidly evolving field of CRISPR-Cas based genome editing and current strategies for extending the capabilities of CRISPR-Cas9, the article also features epigenetic editing, the risks of retinal gene editing, and approaches in development to control Cas9 activity and improve 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, Celia, and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Deputy Chancellor, University of Massachusetts Medical School. (ANI)

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