Study of triple DNA/PEI complexes with anionic peptide coating as vehicles of genetic constructs delivery to muscle tissues

Abstract

Introduction. Skeletal muscles are one of the most complex tissues for transfection, and therefore the development of approaches to the delivery of genetic constructs for the purposes of gene therapy of hereditary neuromuscular diseases (NMD) is relevant.
Aim: the study of DNA complexes/PEI with anionic peptide coating as vehicles of delivering DNA to cells under physiological conditions, as well as to muscle tissues.
Methods. Physicochemical (size, charge, resistance to polyanions), toxic (resazurin test), and transfection (lacZ and GFP gene expression) triple complexes of DNA and polyethylenimine coated with glutamate-rich polypeptides were studied. C57BL/10J mdx mice were used for in vivo experiments.
Results. It has been demonstrated that the developed DNA/PEI/polypeptide complexes are non-toxic, resistant to relaxation caused by polyanions, and can ensure the delivery of genetic constructs to cells and muscle tissues.
Conclusions. The studied triple complexes demonstrated high efficiency of delivering marker genes to muscle tissue and can later be used to deliver gene constructs for the treatment of NMD.