Gene therapy for hemophilia: Current status and future perspectives

Hemophilia is an inherited bleeding disorder caused by the lack of a protein necessary for blood clotting. Gene therapy for hemophilia involves the introduction of a healthy gene into the patient's cells to produce the missing protein.

There are two main types of gene therapy for hemophilia: ex vivo gene therapy, which involves the extraction of cells from the patient, introduction of the healthy gene into these cells in the laboratory, and reintroduction of the modified cells into the patient; and in vivo gene therapy, which involves the direct delivery of the healthy gene into the patient's body using viral vectors.

However, there are still challenges in the large-scale implementation of gene therapy, including safety and long-term efficacy, and the costs associated with the technology.