Ex Vivo COL7A1 Correction for Recessive Dystrophic Epidermolysis Bullosa Using CRISPR/Cas9 and Homology-Directed Repair
Recessive dystrophic epidermolysis bullosa is a rare and severe genetic skin disease resulting in blistering of the skin and mucosa. Recessive dystrophic epidermolysis bullosa (RDEB) is caused by a wide variety of mutations in COL7A1-encoding type VII collagen, which is essential for dermal-epiderma...
Main Authors: | , , , , , |
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Format: | Article |
Language: | English |
Published: |
Elsevier
2018-09-01
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Series: | Molecular Therapy: Nucleic Acids |
Online Access: | http://www.sciencedirect.com/science/article/pii/S2162253118301355 |