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Cas9 protein delivery non-integrating lentiviral vectors for gene correction in sickle cell disease
Gene editing with the CRISPR-Cas9 system could revolutionize hematopoietic stem cell (HSC)-targeted gene therapy for hereditary diseases, including sickle cell disease (SCD). Conventional delivery of editing tools by electroporation limits HSC fitness due to its toxicity; therefore, efficient and no...
Enregistré dans:
| Publié dans: | Mol Ther Methods Clin Dev |
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| Auteurs principaux: | , , , , , , , , , , , |
| Format: | Artigo |
| Langue: | Inglês |
| Publié: |
American Society of Gene & Cell Therapy
2021
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| Sujets: | |
| Accès en ligne: | https://ncbi.nlm.nih.gov/pmc/articles/PMC8005818/ https://ncbi.nlm.nih.gov/pubmed/33816645 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2021.02.022 |
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