abstract |
FIELD: medicine, pharmaceutics. n SUBSTANCE: present invention refers to biotechnology and represents a conjugate used for siRNA intracellular delivery and containing siRNA, which is conjugated by a covalent bond with a hydrophilic compound on one side, e.g. PEG, and with a hydrophobic compound, e.g. cholesterol, on the other side. By self-assembly, the conjugates are able to form homogenous nanoparticles, micellas, wherein the hydrophobic compounds are packed inside the micella; siRNA - between the hydrophobic and hydrophilic compounds, and the hydrophilic compounds - outside. The present invention also discloses methods for producing the above conjugate, pharmaceutical compositions containing the above nanoparticles for the gene therapy of various diseases depending on specific siRNA delivered. What is also disclosed is a pharmaceutical composition for treating cancer, which contains the nanoparticles containing survivin-specific siRNA. n EFFECT: invention enables increasing the siRNA stability in a living body, providing thereby the effective delivery of therapeutic siRNA into cells and the shown activity of siRNA even in a low dose of a relatively low concentration. n 25 cl, 19 dwg, 1 tbl, 6 ex |