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

ABSTRACT:

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 F8 transgene (ET3) with a myeloid-directed CD68 promoter - either without transduction enhancer (group 1) or with transduction enhancer (group 2). Transduced HSCs were transplanted into recipients after myeloablative conditioning. The treatment was assessed for safet

SUBMITTER: Srivastava A 

PROVIDER: S-EPMC11875532 | biostudies-literature | 2025 Jan

REPOSITORIES: biostudies-literature