Genome editing to treat human retinal detachment

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Credit: Mary Ann Liebert, Inc., publishers

New Rochelle, NY, January 19, 2021 – Gene editing therapies, including CRISPR-Cas systems, offer the ability to correct mutations causing hereditary retinal degenerations, a leading cause of blindness. Technological advances in gene preparation, ongoing safety concerns, and strategies to overcome these challenges are highlighted in the peer-reviewed journal. Human gene therapy. Click here to read the free full – text article on the Human gene therapy website.

“Currently, the field is undergoing rapid development with a number of competitive gene editing strategies, including allele-specific downtake, base editing, key editing, and RNA editing, under review. Each offers a different balance of on-target editing. Efficacy against non-target risks, ”state Kanmin Xue, Oxford University, and coauthors. “Testing these newly developed CRISPR technologies in human retinal detachment, organoids and in vivo will help to elucidate the most feasible therapeutic approaches for the treatment of hereditary retinal diseases in the future. coming. “

Highlighting the rapidly changing field of CRISPR-Cas-based genome editing and conventional strategies for expanding CRISPR-Cas9 capabilities, the article also highlights epigenetic editing, retinal gene editing risks, and approaches in development to control Cas9 activity and promote safety.

“The eye is a perfect target for in vivo gene editing. Dr. Xue’s review provides a good insight into the current state of the art,” said the Editor-in-Chief at Human gene therapy Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Chancellor of Dep, University of Massachusetts School of Medicine.

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In Iris

Human gene therapy, The Official Journal of the European Society of Gene and Cell Therapy and eight other international gene therapy associations, the first peer-reviewed journal in the field and providing all-inclusive access to the critical pillars of Human gene therapy: research, methods, and clinical applications. The Journal is led by Editor-in-Chief Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Vice-Chancellor, University of Massachusetts School of Medicine, and internationally renowned editorial board . Human gene therapy available in print and online. Complete tables of content and sample case can be found on the Human gene therapy website.

About the publisher

Mary Ann Liebert Publishers, Inc., is renowned for establishing peer-reviewed authoritative journals in many promising areas of biomedical science and research. His biotechnology trade journal, GEN (Genetic Engineering & Biotechnology News), was the first in its field and is today the most widely read publication in the industry worldwide. A full list of the 90 magazines, books, and news magazines can be found on the website of Mary Ann Liebert, Inc. publishers.

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