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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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| Publié dans: | Mol Ther Methods Clin Dev |
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| Auteurs principaux: | , , , , , , , |
| Format: | Artigo |
| Langue: | Inglês |
| Publié: |
American Society of Gene & Cell Therapy
2020
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| Sujets: | |
| Accès en ligne: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7217921/ https://ncbi.nlm.nih.gov/pubmed/32420407 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2020.04.015 |
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