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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Detalles Bibliográficos
Autores principales: Wrona, Dominik, Pastukhov, Oleksandr, Pritchard, Robert S., Raimondi, Federica, Tchinda, Joëlle, Jinek, Martin, Siler, Ulrich, Reichenbach, Janine
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7217921/
https://www.ncbi.nlm.nih.gov/pubmed/32420407
http://dx.doi.org/10.1016/j.omtm.2020.04.015

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7217921/
https://www.ncbi.nlm.nih.gov/pubmed/32420407
http://dx.doi.org/10.1016/j.omtm.2020.04.015

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