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Enhancing therapeutic efficacy of in vivo platelet-targeted gene therapy in hemophilia A mice
Our previous studies demonstrated that intraosseous (IO) infusion of lentiviral vectors (LVs) carrying a modified B domain–deleted factor VIII (FVIII) transgene driven by a megakaryocyte-specific promoter (GP1Bα promoter; G-F8/N6-LV) successfully transduced hematopoietic stem cells (HSCs) to produce...
Αποθηκεύτηκε σε:
| Τόπος έκδοσης: | Blood Adv |
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| Κύριοι συγγραφείς: | , , , , , , , , , , |
| Μορφή: | Artigo |
| Γλώσσα: | Inglês |
| Έκδοση: |
American Society of Hematology
2020
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| Θέματα: | |
| Διαθέσιμο Online: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7686911/ https://ncbi.nlm.nih.gov/pubmed/33216891 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/bloodadvances.2020002479 |
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