Recombinant adeno-associated virus (rAAV)-mediated expression of a human gamma-globin gene in human progenitor-derived erythroid cells.
Effective gene therapy for the severe hemoglobin (Hb) disorders, sickle-cell anemia and thalassemia, will require an efficient method to transfer, integrate, and express a globin gene in primary erythroid cells. To evaluate recombinant adeno-associated virus (rAAV) for this purpose, we constructed a...
שמור ב:
| הוצא לאור ב: | Proc Natl Acad Sci U S A |
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| Principais autores: | , , , , , |
| פורמט: | Artigo |
| שפה: | Inglês |
| יצא לאור: |
National Academy of Sciences
1994
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| נושאים: | |
| גישה מקוונת: | https://ncbi.nlm.nih.govhttps://pmc.ncbi.nlm.nih.gov/articles/PMC44982/ https://ncbi.nlm.nih.govhttps://pubmed.ncbi.nlm.nih.gov/7524085/ https://ncbi.nlm.nih.govhttps://doi.org/10.1073/pnas.91.21.10183 |
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