FIELD: biotechnology.
SUBSTANCE: described is use of modified RNA containing one open reading frame and modification, which increases expression of coded peptide or protein, wherein said modification includes modification of GC-content in its coding region compared to coding region of its wild type, replacing at least 5 % of substitute codons in a coding region encoding a peptide or protein, thereby increasing the G/C content in said coding region, where the translated amino acid sequence of the wild-type coding region is stored for preparing a therapeutic agent for gene therapy or genetic vaccination, which is introduced by needle-free injection. What is also described is a pharmaceutical composition for gene therapy or genetic vaccination containing modified RNA according to the invention and at least one acceptable pharmaceutical excipient. Claim also describes a pharmaceutical composition and a set of components, which contain said modified RNA to be introduced by jet injection, preferably for use in gene therapy and/or genetic vaccination. In addition, the application describes a method for increasing (localized) expression of encoded RNA peptides or proteins in a dermis or muscle (mammal), consisting in the fact that modified RNA is introduced by means of jet injection. Finally, the application describes a method of treating, comprising administering a modified RNA by a jet injection to an individual in need thereof.
EFFECT: disclosed is a method for increasing expression of encoded RNA proteins.
97 cl, 16 dwg, 4 tbl, 4 ex
Authors
Dates
2020-10-01—Published
2014-08-21—Filed