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Nanoparticles for vaccine and gene therapy: Overcoming the barriers to nucleic acid delivery.
Mollé, Lara M; Smyth, Cameron H; Yuen, Daniel; Johnston, Angus P R.
  • Mollé LM; Drug Delivery, Disposition and Dynamics, Monash Institute of Pharmaceutical Sciences, Monash University, Parkville, Victoria, Australia.
  • Smyth CH; Drug Delivery, Disposition and Dynamics, Monash Institute of Pharmaceutical Sciences, Monash University, Parkville, Victoria, Australia.
  • Yuen D; Drug Delivery, Disposition and Dynamics, Monash Institute of Pharmaceutical Sciences, Monash University, Parkville, Victoria, Australia.
  • Johnston APR; Drug Delivery, Disposition and Dynamics, Monash Institute of Pharmaceutical Sciences, Monash University, Parkville, Victoria, Australia.
Wiley Interdiscip Rev Nanomed Nanobiotechnol ; 14(6): e1809, 2022 11.
Article in English | MEDLINE | ID: covidwho-2170335
ABSTRACT
Nucleic acid therapeutics can be used to control virtually every aspect of cell behavior and therefore have significant potential to treat genetic disorders, infectious diseases, and cancer. However, while clinically approved to treat a small number of diseases, the full potential of nucleic acid therapeutics is hampered by inefficient delivery. Nucleic acids are large, highly charged biomolecules that are sensitive to degradation and so the approaches to deliver these molecules differ significantly from traditional small molecule drugs. Current studies suggest less than 1% of the injected nucleic acid dose is delivered to the target cell in an active form. This inefficient delivery increases costs and limits their use to applications where a small amount of nucleic acid is sufficient. In this review, we focus on two of the major barriers to efficient nucleic acid delivery (1) delivery to the target cell and (2) transport to the subcellular compartment where the nucleic acids are therapeutically active. We explore how nanoparticles can be modified with targeting ligands to increase accumulation in specific cells, and how the composition of the nanoparticle can be engineered to manipulate or disrupt cellular membranes and facilitate delivery to the optimal subcellular compartments. Finally, we highlight how with intelligent material design, nanoparticle delivery systems have been developed to deliver nucleic acids that silence aberrant genes, correct genetic mutations, and act as both therapeutic and prophylactic vaccines. This article is categorized under Nanotechnology Approaches to Biology > Cells at the Nanoscale Therapeutic Approaches and Drug Discovery > Nanomedicine for Infectious Disease Biology-Inspired Nanomaterials > Lipid-Based Structures.
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Full text: Available Collection: International databases Database: MEDLINE Main subject: Vaccines / Nucleic Acids / Communicable Diseases / Nanoparticles Type of study: Prognostic study Topics: Vaccines Limits: Humans Language: English Journal: Wiley Interdiscip Rev Nanomed Nanobiotechnol Year: 2022 Document Type: Article Affiliation country: Wnan.1809

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Full text: Available Collection: International databases Database: MEDLINE Main subject: Vaccines / Nucleic Acids / Communicable Diseases / Nanoparticles Type of study: Prognostic study Topics: Vaccines Limits: Humans Language: English Journal: Wiley Interdiscip Rev Nanomed Nanobiotechnol Year: 2022 Document Type: Article Affiliation country: Wnan.1809