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Ex vivo editing of human hematopoietic stem cells for erythroid expression of therapeutic proteins
Targeted genome editing has a great therapeutic potential to treat disorders that require protein replacement therapy. To develop a platform independent of specific patient mutations, therapeutic transgenes can be inserted in a safe and highly transcribed locus to maximize protein expression. Here,...
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| Publicado en: | Nat Commun |
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| Main Authors: | , , , , , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado: |
Nature Publishing Group UK
2020
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| Assuntos: | |
| Acceso en liña: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7391635/ https://ncbi.nlm.nih.gov/pubmed/32728076 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41467-020-17552-3 |
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