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Annals of Hematology

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11.11.2023 | Review Article

Non-viral and viral delivery systems for hemophilia A therapy: recent development and prospects

verfasst von: Ali Rajabi Zangi, Ala Amiri, Pouya Pazooki, Fatemeh Soltanmohammadi, Hamed Hamishehkar, Yousef Javadzadeh

Erschienen in: Annals of Hematology | Ausgabe 5/2024

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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.

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Titel: Non-viral and viral delivery systems for hemophilia A therapy: recent development and prospects
verfasst von: Ali Rajabi Zangi
Ala Amiri
Pouya Pazooki
Fatemeh Soltanmohammadi
Hamed Hamishehkar
Yousef Javadzadeh
Publikationsdatum: 11.11.2023
Verlag: Springer Berlin Heidelberg
Erschienen in: Annals of Hematology / Ausgabe 5/2024
Print ISSN: 0939-5555
Elektronische ISSN: 1432-0584
DOI: https://doi.org/10.1007/s00277-023-05459-0

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Non-viral and viral delivery systems for hemophilia A therapy: recent development and prospects

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