The bleeding edge: broadening horizons for hematopoietic stem cell therapies.
Trends in molecular medicine
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| Abstract | Recent reports of gene therapy using autologous hematopoietic stem cell transplantation (HSCT) have addressed protein deficiencies of extra-hematopoietic origin. In a recent study, Srivastava et al. report that patients with hemophilia A receiving F8 lentiviral HSCT gene therapy achieved lasting factor VIII restoration and clinical improvement, marking an advance that could enable broader applications of HSCT. |
| Year of Publication | 2025
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| Journal | Trends in molecular medicine
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| Date Published | 03/2025
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| ISSN | 1471-499X
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| DOI | 10.1016/j.molmed.2025.03.003
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| PubMed ID | 40121135
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