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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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| 出版年: | Nat Commun |
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| 主要な著者: | , , , , , , , , , , |
| フォーマット: | Artigo |
| 言語: | Inglês |
| 出版事項: |
Nature Publishing Group UK
2020
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| 主題: | |
| オンライン･アクセス: | 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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