Lentiviral Gene Therapy with CD34+ Hematopoietic Cells for Hemophilia A.

• Tác giả: Fouzia Aboobacker, Aby Abraham, Christopher Benjamin, Harrison C Brown, Rutvi Gautam Dave, Gabriela Denning, Christopher B Doering, Tulasi Geevar, Anbu Jeba, Anu Korula, Uday Kulkarni, Navien Kumaar, Kavitha M Lakshmi, Pete Lollar, Sukesh C Nair, Sushil Selvarajan, Ramachandran V Shaji, Mohana Shankar, Abraham S Singh, Gurbind Singh, Trent Spencer, Alok Srivastava, Vivi Miriam Srivastava
• Ngôn ngữ: eng
• Ký hiệu phân loại: 615.895 Gene therapy
• Thông tin xuất bản: United States : The New England journal of medicine , 2025
• Bộ sưu tập: NCBI
• ID: 751351

Tóm tắt

 BACKGROUND: Severe hemophilia A is managed with factor VIII replacement or hemostatic products that stop or prevent bleeding. Data on gene therapy with hematopoietic stem-cell (HSC)-based expression of factor VIII for the treatment of severe hemophilia A are lacking. METHODS: We conducted a single-center study involving five participants 22 to 41 years of age with severe hemophilia A without factor VIII inhibitors. Autologous HSCs were transduced with CD68-ET3-LV - a lentiviral vector including a new RESULTS: Participants received CD68-ET3-LV-transduced autologous CD34+ HSCs at doses of 5.0×10 CONCLUSIONS: Gene therapy for hemophilia A with the use of lentiviral vector-transduced autologous HSCs resulted in stable factor VIII expression, with factor VIII activity correlating to vector copy number in the peripheral blood. (Funded by the Ministry of Science and Technology, Government of India, and others
  ClinicalTrials.gov number, NCT05265767
  Clinical Trials Registry-India number, CTRI/2022/03/041304.).