Preclinical model for phenotypic correction of dystrophic epidermolysis bullosa by in vivo CRISPR-Cas9 delivery using adenoviral vectors

Preclinical model for phenotypic correction of dystrophic epidermolysis bullosa by in vivo CRISPR-Cas9 delivery using adenoviral vectors

Recessive dystrophic epidermolysis bullosa, a devastating skin fragility disease characterized by recurrent skin blistering, scarring, and a high risk of developing squamous cell carcinoma is caused by mutations in COL7A1, the gene encoding type VII collagen, which is the major component of the anch...

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Bibliographic Details
Main Authors: Marta García, Jose Bonafont, Jesús Martínez-Palacios, Rudan Xu, Giandomenico Turchiano, Stina Svensson, Adrian J. Thrasher, Fernando Larcher, Marcela Del Rio, Rubén Hernández-Alcoceba, Marina I. Garín, Ángeles Mencía, Rodolfo Murillas
Format: Article
Language:English
Published: Elsevier 2022-12-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
gene editing
epidermolysis bullosa
RDEB
adenoviral vector
CRISPR-Cas
in vivo gene therapy
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050122001309

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