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 hemophilia involves introduction healthy gene into patient's cells to produce missing protein. There are two main types hemophilia: ex vivo therapy, which extraction from patient, these in laboratory, and reintroduction modified patient; direct delivery body using viral vectors. However, there still challenges large-scale implementation including safety long-term efficacy, costs associated with technology.