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Gene therapy for hemophilia
Gene therapy offers the potential for a cure for patients with hemophilia by establishing continuous endogenous expression of factor VIII or factor IX (FIX) following transfer of a functional gene to replace the hemophilic patient’s own defective gene. The hemophilias are ideally suited for gene the...
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| Pubblicato in: | Hematology Am Soc Hematol Educ Program |
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| Autore principale: | |
| Natura: | Artigo |
| Lingua: | Inglês |
| Pubblicazione: |
American Society of Hematology
2019
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| Soggetti: | |
| Accesso online: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6913446/ https://ncbi.nlm.nih.gov/pubmed/31808868 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/hematology.2019000007 |
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