This single-center study aimed to evaluate the safety and efficacy of autologous CD34+ hematopoietic stem cells (HSCs) transduced with a lentiviral vector encoding a modified factor VIII transgene in patients with severe Hemophilia A. The objective was to explore a potential long-term therapeutic solution for Hemophilia A through gene therapy.
Methods and Findings:
Five participants aged 22 to 41 years with severe Hemophilia A, without factor VIII inhibitors, were enrolled. Autologous HSCs were transduced with the lentiviral vector and transplanted following myeloablative conditioning. The primary outcomes measured included safety, engraftment, and factor VIII activity levels. The study found that the gene therapy was generally well-tolerated, with sustained factor VIII activity observed in participants, suggesting potential efficacy.
Conclusion:
The findings indicate that lentiviral gene therapy with autologous CD34+ HSCs may offer a promising therapeutic approach for patients with severe Hemophilia A. Further studies with larger cohorts and extended follow-up are necessary to confirm these results and assess long-term safety and efficacy.
