We found a match
Your institution may have access to this item. Find your institution then sign in to continue.
- Title
Non-viral and viral delivery systems for hemophilia A therapy: recent development and prospects.
- Authors
Zangi, Ali Rajabi; Amiri, Ala; Pazooki, Pouya; Soltanmohammadi, Fatemeh; Hamishehkar, Hamed; Javadzadeh, Yousef
- Abstract
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.
- Subjects
HEMOPHILIA; HEMOPHILIA treatment; BLOOD coagulation factor VIII; GENETIC vectors; HEMOPHILIACS
- Publication
Annals of Hematology, 2024, Vol 103, Issue 5, p1493
- ISSN
0939-5555
- Publication type
Article
- DOI
10.1007/s00277-023-05459-0