IMAGE: Provides full access to the critical pillars of human gene therapy: research, clinical methods and applications
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New Rochelle, NEW, 19 January 2021 – Gene editing therapies, including CRISPR-Cas systems, offer the potential to correct mutations causing inherited retinal degeneration, one of the main causes of blindness Technological advances in gene editing, ongoing safety issues 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, the basic edition, the main edition and the ARN edition, are under study. Each offers a different balance between the effectiveness of targeted editing and off-target risks. », declare Kanmin Xue, University of Oxford and its co-authors «Testing these newly developed CRISPR technologies in human retinal tissues, organoids and in vivo will help 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 developing approaches to control Cas9 activity and improve safety

“The eye is an ideal target for in vivo gene editing Dr. Xue's review provides excellent insight into the current state of the art », Says Human Gene Therapy Editor-in-Chief Terence R Flotte, MD, Professor Celia and Isaac Haidak of Medical Education and Dean, provost and director general
Uty Chancellor, University of Massachusetts School of Medicine

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, Clinical methods and applications The Journal is edited by editor Terence R Flotte, MD, Professor Celia and Isaac Haidak of Medical Education and Dean, prévôt et vice-chancelier exécutif, University of Massachusetts School of Medicine, and an esteemed international editorial board Human Gene Therapy is available in print and online Full tables of contents and sample issue are available on the Human Gene Therapy website

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

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Genome modification, retinal degeneration, CRISPR, genetical therapy, genome, retina

News – FR – Modification of the genome to treat human retinal degeneration
Associated title :
Genomic modification to treat human retinal degeneration


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