Matches in SemOpenAlex for { <https://semopenalex.org/work/W2300653550> ?p ?o ?g. }
- W2300653550 endingPage "188" @default.
- W2300653550 startingPage "147" @default.
- W2300653550 abstract "Nucleic acids used to modify the genome or initiate RNA interference (RNAi) including, plasmid DNA (pDNA), messenger RNA (mRNA), short interfering RNA (siRNA), short hairpin RNA (shRNA), microRNA (miRNA), miRNA inhibitors, peptide nucleic acids (PNA), RNA-based adjuvants, and clustered regularly interspaced short palindromic repeats/Cas (CRISPR/Cas) gene-editing systems have powerful therapeutic potential to modulate currently undruggable targets and to provide improved treatment options for the most challenging pathologies. In all cases, the efficacy of these nucleic acid drugs is contingent upon effective delivery to the organ, cell population, and intracellular compartment of interest. Rapid advances in polymer chemistry and materials science have made it possible to rationally design non-viral nucleic acid vectors which can overcome delivery barriers and increase the efficacy and specificity of gene expression, editing, or inhibition. Herein, we review five distinct classes of polymeric nanoparticles which have been heavily investigated for gene delivery: micelles/polyplexes, cross-linked micelles, polymersomes, microgels, and nanogels. These different classes of polymer delivery systems provide virus mimetic functionalities such as nucleic acid protection, increased blood circulation time, active and passive targeting, evasion of immune system, cellular internalization, and endolysosomal escape to varying extents. Recent studies are reviewed in order to showcase the potential for success within each class, and current ongoing clinical trials for RNAi are discussed briefly to highlight the translatability of optimized nanoparticle designs." @default.
- W2300653550 created "2016-06-24" @default.
- W2300653550 creator A5041525182 @default.
- W2300653550 creator A5075794128 @default.
- W2300653550 date "2016-01-01" @default.
- W2300653550 modified "2023-10-18" @default.
- W2300653550 title "Polymeric nanoparticles for gene delivery" @default.
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