Highly efficient ex-vivo correction of COL7A1 through RNP-based CRISPR/Cas9 and Homology-Directed Repair to treat recessive dystrophic epidermolysis bullosa-Reference-Cited by-同舟云学术

Highly efficient ex-vivo correction of COL7A1 through RNP-based CRISPR/Cas9 and Homology-Directed Repair to treat recessive dystrophic epidermolysis bullosa

Published:2023-12 Issue: Volume: Page:
ISSN:0022-202X
Container-title:Journal of Investigative Dermatology
language:en
Short-container-title:Journal of Investigative Dermatology

Author:

Berthault Camille^ORCID,Gaucher Sonia^ORCID,Gouin Olivier^ORCID,Schmitt Alain^ORCID,Chen Mei^ORCID,Woodley David^ORCID,Titeux Matthias^ORCID,Hovnanian Alain^ORCID,Izmiryan Araksya^ORCID

Funder

European Research Council

Institut National de la Santé et de la Recherche Médicale

Dystrophic Epidermolysis Bullosa Research Association

DEBRA International

Publisher

Elsevier BV

Subject

Cell Biology,Dermatology,Molecular Biology,Biochemistry

Reference38 articles.

1. Correction of recessive dystrophic epidermolysis bullosa by homology-directed repair-mediated genome editing. Molecular therapy;Bonafont;the journal of the American Society of Gene Therapy,2021

2. Clinically Relevant Correction of Recessive Dystrophic Epidermolysis Bullosa by Dual sgRNA CRISPR/Cas9-Mediated Gene Editing. Molecular therapy;Bonafont;the journal of the American Society of Gene Therapy,2019

3. Challenges of Gene Editing Therapies for Genodermatoses;Brooks;International journal of molecular sciences 24,2023

4. Multiplex genome engineering using CRISPR/Cas systems;Cong;Science,2013

5. High frequency of the 425A-->G splice-site mutation and novel mutations of the COL7A1 gene in central Europe: significance for future mutation detection strategies in dystrophic epidermolysis bullosa;Csikos;The British journal of dermatology,2005