Gene therapy with hematopoietic cells for hemophilia A
Haemophilia A is a blood clotting disorder caused by a genetic deficiency in clotting factor VIII, thereby resulting in significant susceptibility to bleeding, both internally and externally.
This condition occurs almost exclusively in males born to carrier mothers due to X-linked recessive inheritance.
Recent reports of gene therapy using autologous hematopoietic stem cell transplantation (HSCT) have addressed protein deficiencies of extra-hematopoietic origin.
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.
In a recent study, the researchers 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.
https://www.cell.com/trends/molecular-medicine/abstract/S1471-4914(25)00054-1
https://sciencemission.com/hematopoietic-stem-cell-therapies-for-hemophilia
