Student Research Committee, Tabriz University of Medical Sciences, Tabriz, Iran.
Department of Pharmaceutics, Faculty of Pharmacy, Tabriz University of Medical Sciences, Tabriz, 5166-15731, Iran.
Ann Hematol. 2024 May;103(5):1493-1511. doi: 10.1007/s00277-023-05459-0. Epub 2023 Nov 11.
Recent advancements have focused on enhancing factor VIII half-life and refining its delivery methods, despite the well-established knowledge that factor VIII deficiency is the main clotting protein lacking in hemophilia. Consequently, both viral and non-viral delivery systems play a crucial role in enhancing the quality of life for hemophilia patients. The utilization of viral vectors and the manipulation of non-viral vectors through targeted delivery are significant advancements in the field of cellular and molecular therapies for hemophilia. These developments contribute to the progression of treatment strategies and hold great promise for improving the overall well-being of individuals with hemophilia. This review study comprehensively explores the application of viral and non-viral vectors in cellular (specifically T cell) and molecular therapy approaches, such as RNA, monoclonal antibody (mAb), and CRISPR therapeutics, with the aim of addressing the challenges in hemophilia treatment. By examining these innovative strategies, the study aims to shed light on potential solutions to enhance the efficacy and outcomes of hemophilia therapy.
最近的进展集中在提高因子 VIII 的半衰期和改进其给药方法上,尽管人们已经清楚地认识到,因子 VIII 缺乏是血友病中缺乏的主要凝血蛋白。因此,病毒和非病毒递送系统在提高血友病患者生活质量方面都起着至关重要的作用。病毒载体的利用和通过靶向递送对非病毒载体的操纵是血友病细胞和分子治疗领域的重大进展。这些发展有助于推进治疗策略的进展,并为改善血友病患者的整体健康状况带来了巨大的希望。本综述研究全面探讨了病毒和非病毒载体在细胞(特别是 T 细胞)和分子治疗方法中的应用,如 RNA、单克隆抗体(mAb)和 CRISPR 治疗,以解决血友病治疗中的挑战。通过研究这些创新策略,本研究旨在探讨提高血友病治疗效果的潜在解决方案。