Efficient Dual Cas9 Nickase Correction of a Prevalent Pathogenic LAMB3 Variant for Junctional Epidermolysis Bullosa

Efficient Dual Cas9 Nickase Correction of a Prevalent Pathogenic LAMB3 Variant for Junctional Epidermolysis Bullosa

Gene editing facilitated by homology-directed repair represents a promising strategy for precisely correcting pathogenic variants underlying monogenic disorders, including the life-threatening skin blistering condition junctional epidermolysis bullosa (JEB). Frequent reports of unintended off-target...

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Bibliographic Details
Main Authors: Alex du Rand, John Hunt, Daniel Verdon, Ben Buttle, P. Rod Dunbar, Diana Purvis, Vaughan Feisst, Hilary Sheppard
Format: Article
Language:English
Published: Elsevier 2025-05-01
Series:JID Innovations
Subjects:
CRISPR/Cas9
Gene correction
Gene therapy
Homology-directed repair
Skin grafts
Online Access:http://www.sciencedirect.com/science/article/pii/S2667026724000912
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