Article
Oral gene therapy for hemophilia B using chitosan-formulated FIX mutants.
Journal of thrombosis and haemostasis : JTH - 1 Jun 2014
Quade-Lyssy P, Milanov P, Abriss D, Ungerer C, Königs C, Seifried E, Schüttrumpf J
Abstract excerpt
BACKGROUND: Oral gene delivery of non-viral vectors is an attractive strategy to achieve transgene expression. Although expected efficacy from non-viral delivery systems is relatively low, repeated vector administration is possible and may help to obtain durable transgene expression in a therapeutic range. OBJECTIVES: To test the principle feasibility of using factor (F) IX variants with improved function...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.
