Lentiviral Gene Therapy with CD34+ Hematopoietic Cells for Hemophilia A
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.).
Authors
- Aby Abraham (ORCID: https://orcid.org/0000-0002-2596-4434)
- Majji Shankar (ORCID: https://orcid.org/0000-0003-3758-9069)
- Christopher B. Doering (ORCID: https://orcid.org/0000-0002-5662-0060)
- Alok Srivastava
- Abraham Sunder Singh
- Anu Korula (ORCID: https://orcid.org/0000-0003-3207-3370)
- Pete Lollar (ORCID: https://orcid.org/0000-0002-1206-8104)
- Rutvi Gautam Dave (ORCID: https://orcid.org/0000-0002-1294-7061)
- Sushil Selvarajan (ORCID: https://orcid.org/0000-0003-4045-3496)
- Gabriela Denning
- Harrison C. Brown
- Tulasi Geevar (ORCID: https://orcid.org/0000-0002-4247-4154)
- Shaji R Velayudhan (ORCID: https://orcid.org/0000-0002-8600-2933)
- Uday Kulkarni (ORCID: https://orcid.org/0000-0003-2230-2424)
- H. Trent Spencer (ORCID: https://orcid.org/0000-0001-8062-2202)
- Anbu Jeba
- Kavitha M. Lakshmi (ORCID: https://orcid.org/0000-0002-7870-8020)
- Christopher Benjamin
- Vivi M Srivastava (ORCID: https://orcid.org/0000-0001-6404-3622)
- Sukesh C. Nair
- Gurbind Singh
- Fouzia N. Aboobacker
- Navien Kumaar
Institutions
- Christian Medical College, Vellore (IN)
Publication Details
- Journal
- New England Journal of Medicine
- Published
- 2024-12-10
- DOI
- https://doi.org/10.1056/nejmoa2410597
- Citations
- 42
- Primary Topic
- Hemophilia Treatment and Research
- Type
- article
- Field-Weighted Citation Impact
- 19.08
Funders
- Hemophilia of Georgia
- Department of Biotechnology, Ministry of Science and Technology, India
- National Heart, Lung, and Blood Institute