Hemophilia is a hereditary disorder that can be life-threatening in individuals who have severe spontaneous bleeding resulting from minor trauma or surgery. Although replacement therapy of the missing exogenous factor has improved patients’ quality of life, it has not been possible to establish a long-term treatment. Due to the severity of the disease and the need for repetitive doses throughout the patient’s life, replacement therapy has become a high-cost treatment option; therefore, the development of self-sustainable long-term therapies is critical. Hemophilia is a good candidate for gene therapy because it is a monogenic disease that can be counteracted by expression of the missing factor. In this article, we review some of the most relevant advances in gene therapy for this illness.

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血友病基因治疗进展

血友病是一种遗传性疾病，对于因轻微创伤或手术导致严重自发性出血的个体，可能会危及生命。虽然缺失外源性因素的替代疗法改善了患者的生活质量，但尚无法建立长期治疗。由于疾病的严重性以及患者一生需要重复给药，替代疗法已成为一种高成本的治疗选择；因此，开发可自我维持的长期疗法至关重要。血友病是基因治疗的良好候选者，因为它是一种单基因疾病，可以通过表达缺失因子来抵消。在本文中，我们回顾了该疾病基因治疗的一些最相关的进展。