» Articles » PMID: 39356099

Polymeric Vehicles for Nucleic Acid Delivery: Enhancing the Therapeutic Efficacy and Cellular Uptake

Overview
Specialty Pharmacology
Date 2024 Oct 2
PMID 39356099
Authors
Affiliations
Soon will be listed here.
Abstract

Background: Therapeutic gene delivery may be facilitated by the use of polymeric carriers. When combined with nucleic acids to form nanoparticles or polyplexes, a variety of polymers may shield the cargo from in vivo breakdown and clearance while also making it easier for it to enter intracellular compartments.

Aim And Objectives: Polymer synthesis design choices result in a wide variety of compounds and vehicle compositions. Depending on the application, these characteristics may be changed to provide enhanced endosomal escape, longer-lasting distribution, or stronger connection with nucleic acid cargo and cells. Here, we outline current methods for delivering genes in preclinical and clinical settings using polymers.

Methodology: Significant therapeutic outcomes have previously been attained using genetic material- delivering polymer vehicles in both in-vitro and animal models. When combined with nucleic acids to form nanoparticles or polyplexes, a variety of polymers may shield the cargo from in vivo breakdown and clearance while also making it easier for it to enter intracellular compartments. Many innovative diagnoses for nucleic acids have been investigated and put through clinical assessment in the past 20 years.

Results: Polymer-based carriers have additional delivery issues due to their changes in method and place of biological action, as well as variances in biophysical characteristics. We cover recent custom polymeric carrier architectures that were tuned for nucleic acid payloads such genomemodifying nucleic acids, siRNA, microRNA, and plasmid DNA.

Conclusion: In conclusion, the development of polymeric carriers for gene delivery holds promise for therapeutic applications. Through careful design and optimization, these carriers can overcome various challenges associated with nucleic acid delivery, offering new avenues for treating a wide range of diseases.

References
1.
TATUM E . Molecular biology, nucleic acids, and the future of medicine. Perspect Biol Med. 1966; 10(1):19-32. DOI: 10.1353/pbm.1966.0027. View

2.
Gibney E, Nolan C . Epigenetics and gene expression. Heredity (Edinb). 2010; 105(1):4-13. DOI: 10.1038/hdy.2010.54. View

3.
Dunbar C, High K, Joung J, Kohn D, Ozawa K, Sadelain M . Gene therapy comes of age. Science. 2018; 359(6372). DOI: 10.1126/science.aan4672. View

4.
Orkin S, Reilly P . MEDICINE. Paying for future success in gene therapy. Science. 2016; 352(6289):1059-61. DOI: 10.1126/science.aaf4770. View

5.
Zhao Y, Huang L . Lipid nanoparticles for gene delivery. Adv Genet. 2014; 88:13-36. PMC: 5006671. DOI: 10.1016/B978-0-12-800148-6.00002-X. View