Intravenous Delivery of piggyBac Transposons as a Useful Tool for Liver-Specific Gene-Switching
نویسندگان
چکیده
منابع مشابه
Hyperactive PiggyBac Transposons for Sustained and Robust Liver-targeted Gene Therapy
The development of robust nonviral vectors could facilitate clinical gene therapy applications and may overcome some of the immune complications of viral vectors. Nevertheless, most nonviral gene deliver approaches typically yield only transient and/or low gene expression. To address these caveats, we have explored piggyBac transposons to correct hemophilia B by liver-directed factor IX (FIX) g...
متن کاملHyperactive Piggybac Transposons for Sustained and Robust Liver-targeted Gene Therapy Authors
Department of Gene Therapy & Regenerative Medicine, Free University of Brussels, Brussels, Belgium & Center for Molecular & Vascular Biology, Department of Cardiovascular Medicine, University of Leuven, Leuven, Belgium; Molecular Immunology Unit, Institute of Child Health, University College London, London, United Kingdom; Institute for Women’s Health Gene Transfer Technology Group, University ...
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O of the more intractable problems in gene therapy over the past decade has been the development of methods to allow the selective delivery of genes to the liver and their stable maintenance and expression therein. Hepatocytes (the principal parenchymal cell of the liver) are a much sought-after target of gene therapy because they play many unique roles in the physiology of the mammalian host (...
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ژورنال
عنوان ژورنال: International Journal of Molecular Sciences
سال: 2018
ISSN: 1422-0067
DOI: 10.3390/ijms19113452