Gene therapy for hemophilia: Recent developments and challenges (Review)

 Hemophilia is an X‑linked disease marked by the absence or impaired function of specific coagulation factors. Hemophilia A is characterized by a deficiency in factor VIII, and hemophilia B is characterized by a deficiency in factor IX. Factor XI deficiency, referred to as hemophilia C, is a very uncommon bleeding illness characterized by moderate symptoms; although the condition is not widespread, standard management poses significant challenges linked to socioeconomic factors. The introduction of FDA‑approved gene therapy for hemophilia B in 2022 holds transformative potential; however, certain issues demand careful consideration. The present review provides insight into the existing hemophilia landscape, discusses the latest strides in gene therapy for hemophilia, and examines the hurdles that need to be addressed to make this innovative treatment more accessible to individuals afflicted by this condition.