CRISPR-Directed Therapeutic Correction at the NCF1 Locus Is Challenged by Frequent Incidence of Chromosomal Deletions

CRISPR-Directed Therapeutic Correction at the NCF1 Locus Is Challenged by Frequent Incidence of Chromosomal Deletions

Resurrection of non-processed pseudogenes may increase the efficacy of therapeutic gene editing, upon simultaneous targeting of a mutated gene and its highly homologous pseudogenes. To investigate the potency of this approach for clinical gene therapy of human diseases, we corrected a pseudogene-ass...

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Bibliographic Details
Main Authors: Dominik Wrona, Oleksandr Pastukhov, Robert S. Pritchard, Federica Raimondi, Joëlle Tchinda, Martin Jinek, Ulrich Siler, Janine Reichenbach
Format: Article
Language:English
Published: Elsevier 2020-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
chronic granulomatous disease
CGD
gene editing
CRISPR
NCF1
pseudogene
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050120300759

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http://www.sciencedirect.com/science/article/pii/S2329050120300759

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