Журнал генетических синдромов и генной терапии
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Advancements in Non Viral Gene Therapy for Hemophilia

Bradley S. Fletcher

Over the last 10-15 years, significant advances in vector design and delivery techniques have facilitated the development of nonviral approaches for the treatment of hemophilia. Despite these advancements, there remain several obstacles preventing the successful application of these approaches in larger mammals such as dogs and humans. This review covers nonviral gene therapy approaches using both in vivo gene delivery and ex vivo gene transfer. Plasmid-based approaches, as well as integrating transposons are examined for efficacy, risks and limitations. Results are presented on the only human clinical trial in hemophilia that utilized nonviral approaches.