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In vivo hematopoietic stem cell gene therapy ameliorates murine thalassemia intermedia
Current thalassemia gene therapy protocols require the collection of hematopoietic stem/progenitor cells (HSPCs), in vitro culture, lentivirus vector transduction, and retransplantation into myeloablated patients. Because of cost and technical complexity, it is unlikely that such protocols will be a...
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| 出版年: | J Clin Invest |
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| 主要な著者: | , , , , , , , , , , , |
| フォーマット: | Artigo |
| 言語: | Inglês |
| 出版事項: |
American Society for Clinical Investigation
2018
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| 主題: | |
| オンライン･アクセス: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6355219/ https://ncbi.nlm.nih.gov/pubmed/30422819 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/JCI122836 |
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