Abstract
Materials for targeted drug delivery and gene transfer, in particular for DNA and siRNA delivery into cells, are currently the two most relevant in the international pharmacological market. The leading positions in the field of drug delivery belong to the biocompatible and biodegradable multifunctional nanoscale materials capable of forming water-soluble forms of drugs, providing their addressed delivery, and crossing the biological barriers in the organism. Labeling these materials with a biocompatible dye is also important for monitoring drug action or gene expression, biodistribution, and excretion from the treated organism.
In this chapter, principal approaches used in the synthesis of materials for gene delivery are shown, and their biocompatibility and functions are characterized for cells of different origin, namely, bacteria, yeast, plants, and mammals. Usually, the DNA that should be delivered to the target cells is “labeled” by gene coding for the green fluorescent protein (GFP); the gene delivery platform might be additionally labeled with a specific fluorescent dye. This helps monitoring the uptake, biodistribution, and location of the action of the transported nucleic acid in the cell (tissue, organ, organism), as well as the ways of possible clearance of the gene delivery platform from the body.
The chemical structure of materials for delivery of nucleic acids used in gene therapy should contain a positively charged group, usually an amine. Some advanced platforms also contain covalently conjugated vector elements (e.g., antibody or other ligand for specific receptors on plasma membrane of the targeted cells) for the addressed action. The hybrid platforms for simultaneous delivery of anticancer drugs and specific siRNA were created, and such multifunctionality is an important trend in the development of novel nanoscale materials used for disease treatment, gene therapy, and other biomedical purposes.
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Abbreviations
- AEM:
-
aminoethyl methacrylate
- BA :
-
butyl acrylate
- CMV:
-
cytomegalovirus
- DEAE:
-
diethylethanolamine
- DMAEMA:
-
(2-dimethylamino)ethyl methacrylate
- DNA :
-
deoxyribonucleic acid
- F8MA:
-
octafluoropentyl methacrylate
- FA:
-
folic acid
- GARD :
-
Genetic and Rare Diseases Information Center
- GFP :
-
Green Fluorescent Protein
- GMA :
-
glycidyl methacrylate
- GO :
-
graphene oxide
- LB media :
-
Luria-Bertani media
- LMA :
-
lauryl methacrylate
- MA:
-
maleic acid
- miRNA :
-
microRNA
- MP :
-
1-isopropyl-3(4)-[1-(tert-butyl peroxy)-1-methylethyl] benzene
- mPEG :
-
poly(ethylene glycol) methyl ether
- mRNA :
-
messenger ribonucleic acid
- NCATS :
-
National Center for Advancing Translational Sciences
- NVP :
-
N-vinylpyrrolidone
- PAH :
-
polyallylamine hydrochloride
- pDNA:
-
plasmid DNA
- PEG:
-
poly(ethylene glycol)
- PEGMA:
-
poly(ethylene glycol)monomethyl ether methacrylate
- PEI:
-
polyethyleneimine
- pHEMA :
-
poly(2-hydroxyethyl methacrylate)
- PLGA:
-
poly(lactide-co-glycolide)
- siRNA:
-
small interfering ribonucleic acid
- VA:
-
vinyl acetate
- VEP :
-
5-tert butylperoxy-5-methyl-l-hexene-3-yne.
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Acknowledgements
The research was supported by the Research Grant of the National Academy of Sciences of Ukraine to research laboratories/groups of young scientists of the National Academy of Sciences of Ukraine (# 0120U100197, 2020–2021), and Research Grant of the Molecular & Cellular Biotechnologies Program of the National Academy of Sciences of Ukraine (# 0115U004198, 2015–2019). The authors sincerely thank Olga Klyuchivska (Institute of Cell Biology, National Academy of Sciences of Ukraine, Lviv, Ukraine) for technical support at fluorescent microscopy experiments, and Dr. Bodnar L.S. (Department of Genetics and Biotechnology, Ivan Franko National University of Lviv, Lviv, Ukraine) for consultation and technical support at using Ames test.
This work was also supported by Cedars-Sinai Medical Center’s International Research and Innovation in Medicine Program and the Association for Regional Cooperation in the Fields of Health, Science and Technology (RECOOP HST) Association.
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Finiuk, N., Mitina, N., Zaichenko, A., Stoika, R. (2022). Bioimaging, Biocompatibility, and Functioning of Polymeric Nanocarriers for Gene Delivery. In: Stoika, R.S. (eds) Biomedical Nanomaterials. Springer, Cham. https://doi.org/10.1007/978-3-030-76235-3_8
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