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IMAGE: Provides all-inclusive access to the critical pillars of human gene therapy: research, methods, and clinical applications.
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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 potential to correct mutations causing inherited retinal degenerations, a leading cause of blindness. Technological advances in gene editing, continuing 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 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 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, ....

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