| Literature DB >> 31063396 |
Keith Henry Moss1, Petya Popova2, Sine R Hadrup1, Kira Astakhova2, Maria Taskova2.
Abstract
Gene therapy is an exciting field that has the potential to address emerging scientific and therapeutic tasks. RNA-based gene therapy has made remarkable progress in recent decades. Nevertheless, efficient targeted delivery of RNA therapeutics is still a prerequisite for entering the clinics. In this review, we introduce current delivery methods for RNA gene therapeutics based on lipid nanoparticles (LNPs). We focus on the clinical appeal of recent RNA NPs and discuss existing challenges of fabrication and screening LNP candidates for effective translation into drugs of human metabolic diseases and cancer.Entities:
Keywords: LNP fabrication; LNP screening; RNA; delivery; gene therapy; lipid nanoparticle (LNP)
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Year: 2019 PMID: 31063396 DOI: 10.1021/acs.molpharmaceut.8b01290
Source DB: PubMed Journal: Mol Pharm ISSN: 1543-8384 Impact factor: 4.939