International Journal of Nanomedicine (Aug 2021)

Recent Advances and Challenges in Gene Delivery Mediated by Polyester-Based Nanoparticles

  • Piperno A,
  • Sciortino MT,
  • Giusto E,
  • Montesi M,
  • Panseri S,
  • Scala A

Journal volume & issue
Vol. Volume 16
pp. 5981 – 6002

Abstract

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Anna Piperno,1 Maria Teresa Sciortino,1 Elena Giusto,2 Monica Montesi,2 Silvia Panseri,2 Angela Scala1 1Department of Chemical, Biological, Pharmaceutical and Environmental Sciences, University of Messina, Messina, Italy; 2CNR-ISTEC, Institute of Science and Technology for Ceramics, National Research Council of Italy, Faenza, RA, ItalyCorrespondence: Angela ScalaDepartment of Chemical, Biological, Pharmaceutical and Environmental Sciences, University of Messina, Messina, ItalyTel +39 090 6765515Fax +39 090 393897Email [email protected]: Gene therapy is a promising approach for the treatment of several diseases, such as chronic or viral infections, inherited disorders, and cancer. The cellular internalization of exogenous nucleic acids (NA) requires efficient delivery vehicles to overcome their inherent pharmacokinetic drawbacks, e.g. electrostatic repulsions, enzymatic degradation, limited cellular uptake, fast clearance, etc. Nanotechnological advancements have enabled the use of polymer-based nanostructured biomaterials as safe and effective gene delivery systems, in addition to viral vector delivery methods. Among the plethora of polymeric nanoparticles (NPs), this review will provide a comprehensive and in-depth summary of the polyester-based nanovehicles, including poly(lactic-co-glycolic acid) (PLGA) and polylactic acid (PLA) NPs, used to deliver a variety of foreign NA, e.g. short interfering RNA (siRNA), messenger RNA (mRNA), and plasmid DNA (pDNA). The article will review the versatility of polyester-based nanocarriers including their recent application in the delivery of the clustered, regularly‐interspaced, short palindromic repeats/Cas (CRISPR/Cas) genome editing system for treating gene-related diseases. The remaining challenges and future trend of the targeted delivery of this revolutionary genome-editing system will be discussed. Special attention will be given to the pivotal role of nanotechnology in tackling emerging infections such as coronavirus disease 2019 (COVID-19): ground-breaking mRNA vaccines delivered by NPs are currently used worldwide to fight the pandemic, pushing the boundaries of gene therapy.Keywords: PLGA, PLA, siRNA, CRISPR, COVID-19, CLAN

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