Study of electrostatically stabilized nucleopeptide complexes for DNA delivery into muscle cells
https://doi.org/10.25557/2073-7998.2025.10.80-83
Abstract
Introduction. Symptomatic therapy for Duchenne muscular dystrophy is unable to prevent the development of the disease, so hopes for treating the disease are concentrated in the field of gene therapy, which allows compensating for genetic defects by introducing nucleic acids into the cell.
Aim: study of the properties of cationic and anionic peptides to improve the DNA delivery to muscle cells.
Methods. Methods of C2C12 myoblast cultivation, cell transfection, analysis of toxic properties using a resazurin test, and flow cytofluorometry were used in the work. C57BL/10Jmdx mice were used for in vivo experiments.
Results. Most of the developed complexes are not toxic to C2C12 myoblasts and effectively deliver plasmid DNA to muscle cells both in vitro and in vivo.
Conclusions. The most effective was the nucleopeptide complex containing the largest amount of shielding coating in its composition, which increases its stability in the extracellular matrix of skeletal muscles and, thus, ensures the greatest efficiency of DNA delivery.
Keywords
About the Authors
A. A. EgorovaRussian Federation
3, Mendeleevskaya line, St. Petersburg, 199034
I. Y. Kogan
Russian Federation
3, Mendeleevskaya line, St. Petersburg, 199034
A. V. Kiselev
Russian Federation
3, Mendeleevskaya line, St. Petersburg, 199034
References
1. Guiraud S., Aartsma-Rus A,. Vieira N.M., et al. The Pathogenesis and Therapy of Muscular Dystrophies. Annu Rev Genomics Hum Genet. 2015;16:281-308.
2. Cohen S.A., Bar-Am O., Fuoco C. et al. In vivo restoration of dystrophin expression in mdx mice using intra-muscular and intraarterial injections of hydrogel microsphere carriers of exon skipping antisense oligonucleotides. Cell Death and Disease. 2022;13: 779.
3. McDonald C.M., Campbell C., Torricelli R.E., et al. Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebocontrolled, phase 3 trial. Lancet. 2017;390(10101):1489-1498.
4. Lu Q.L., Bou-Gharios G., Partridge T.A. Non-viral gene delivery in skeletal muscle: A protein factory. Gene Therapy. 2003; 10(2): 131– 142.
5. Jiao Y., Xia Z.L., Ze L.J., et al. Research Progress of nucleic acid delivery vectors for gene therapy. Biomed Microdevices. 2020;22(1):16.
6. Egorova A.A., Kiselev A.V. Peptide modules for overcoming barriers of nucleic acids transport to cells. Current Topics in Medicinal Chemistry. 2016;16(3):330–342.
7. McErlean E.M., McCrudden C.M., McCarthy H.O. Delivery of nucleic acids for cancer gene therapy: Overcoming extra-and intracellular barriers. Therapeutic Delivery. 2016;7(9):619–637.
Review
For citations:
Egorova A.A., Kogan I.Y., Kiselev A.V. Study of electrostatically stabilized nucleopeptide complexes for DNA delivery into muscle cells. Medical Genetics. 2025;24(10):80-83. (In Russ.) https://doi.org/10.25557/2073-7998.2025.10.80-83






















