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Study highlights genome altering can help to treat human retinal degeneration ANI | Updated: Jan 20, 2021 09:14 IST
New York [US], January 20 (ANI): A study published in journal Human Gene Therapy shows that gene editing therapies, including CRISPR-Cas frameworks, offer the possibility to address transformations causing inherited retinal degenerations, a leading cause of blindness.
The findings highlight the technological advances in gene editing, continuing safety concerns, and strategies to overcome these challenges.
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.
Gene editing therapies, including CRISPR-Cas systems, offer the potential to correct mutations causing inherited retinal degenerations, a leading cause of blindness.
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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, state Kanmin Xue, Unive