GEN-SPECIFIC SIRNA RELATED TO RESPIRATORY DISEASE, TWO-SPIRAL CONSTRUCT OF OLIGO-RNA COMPRISING SIRNA, AND CONTAINING IT COMPOSITION FOR PREVENTION OR TREATMENT OF RESPIRATORY DISEASE Russian patent published in 2018 - IPC C12N15/113 A61K48/00 A61P11/00 

FIELD: biotechnology.

SUBSTANCE: construct and a nanoparticle containing the specified construct are provided for efficient delivering a CTGF-specific siRNA to cells, as well as a pharmaceutical composition and a compound comprising the said lyophilized pharmaceutical composition for preventing or treating respiratory diseases, and a method for preventing or treating respiratory diseases. Specified construct comprises a hydrophilic and a hydrophobic materials directly coupled through a single covalent bond or by linkers with different ends of the sense or the antisense strand of the CTGF-specific siRNA. Structure of the proposed construct provides spontaneous assembling of nanoparticles containing the said construct, which have low polydispersity indices and uniform dimensions. Control over the structure of the hydrophilic fragment of the proposed construct provides the possibility of regulating the polydispersity index and the size of the nanoparticles.

EFFECT: proposed group of inventions provides efficient delivery of the CTGF-specific siRNA to cells and can be used in medicine to prevent or treat respiratory diseases.

23 cl, 25 dwg, 2 tbl, 11 ex