Modification of the genome to treat human retinal degeneration

New Rochelle, NY, January 19, 2021 – Gene-editing therapies, including CRISPR-Cas systems, have the potential to correct mutations causing inherited retinal degeneration, one of the leading causes of blindness. Technological advancements in gene editing, persistent safety concerns, and strategies to overcome these challenges are highlighted in the peer-reviewed journal Human gene therapy. Click here to read the full text article for free on the Human gene therapy website.

“Currently, the field is undergoing rapid development with a number of competing gene editing strategies, including allele-specific knockdown, basic editing, major editing and editing. of RNA, are under study. Each offers a different balance of on-target efficiency versus off-target risk, ”say Kanmin Xue, University of Oxford and co-authors. “Testing these newly developed CRISPR technologies in human retinal tissue, organoids and in vivo will help to highlight the most viable therapeutic approaches for the treatment of inherited retinal diseases in the future.

Characterizing the rapidly evolving field of CRISPR-Cas-based genome editing and current strategies for expanding the capabilities of CRISPR-Cas9, the article also presents 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 gene editing in vivo. 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, Professor Celia and Isaac Haidak of Medical Education and Dean, Provost and Executive Assistant Chancellor of the University of Massachusetts Medical School.

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Human gene therapy, the Official Journal of the European Society for Gene and Cell Therapy and eight other international gene therapy societies, was the first peer-reviewed journal in the field and provides full access to the critical pillars of Human gene therapy: research, methods and clinical applications. The Journal is edited by Editor-in-Chief Terence R. Flotte, MD, Professor Celia and Isaac Haidak in Medical Education and Dean, Provost and Executive Vice Chancellor of the University of Massachusetts Medical School, and an Editorial Board international estimated. Human gene therapy is available in print and online. Full tables of contents and a sample number are available on the Human gene therapy website.

Mary Ann Liebert, Inc., editors are known for creating authoritative peer-reviewed journals in many promising areas of biomedical science and research. Its biotechnology magazine, GEN (Genetic Engineering & Biotechnology News), was the first in its field and is today the most widely read industry publication in the world. A complete list of the company’s 90 journals, books, and news magazines is available on the Mary Ann Liebert, Inc. editors website.