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Published on: March 16, 2022
Gene therapy progress and prospects: non-viral gene therapy by systemic delivery
1Division of Molecular Pharmaceutics, School of Pharmacy, University of North Carolina, Chapel Hill, NC 27599-7360, USA.
Abstract:
Non-viral vectors continue to be an attractive alternative to viral vectors due to their safety, versatility and ease of preparation and scale-up. Over the past few years, investigators have been successful in developing gene carriers that can be targeted to the disease site. Several different delivery vectors for systemic use have been developed by different groups for plasmid DNA and oligonucleotide. Most of them are designed for targeted tumor therapy. The mechanism of inflammatory toxicity, the major toxicity of cationic lipoplex, has been studied and managed. In this review, we focus on the progress made over the last 2 years. We also discuss some future prospects for gene delivery.
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