FIELD: medicine, pharmaceutics.
SUBSTANCE: invention refers to molecular biology, genetics and medicine. What is presented is a lipid modified double-stranded RNA comprising an antisense chain having a nucleotide sequence complementary to a target sequence, and a sense chain having a nucleotide sequence complementary to the antisense chain. The sense chain comprises a lipid attached either directly or via a linker to at least one of the first six nucleotides of the 5'-terminal end. The given double-stranded RNA can inhibit expression of the target gene. The presented RNA possesses highly nuclease resistance, intracellular accumulation effectiveness and a high effect of RNA interference.
EFFECT: invention may be used in medicine for treating the diseases mediated by genetic disorders.
12 cl, 10 dwg, 2 ex
| Title | Year | Author | Number |
|---|---|---|---|
| TWO-STRANDED RNA MODIFIED WITH LIPIDS AND HAVING POWERFUL EFFECT OF RNA INTERFERENCE | 2009 |
|
RU2501859C2 |
| SINGLE-STRANDED CIRCULAR RNA AND METHOD FOR PRODUCING IT | 2008 |
|
RU2523596C2 |
| CHEMICALLY MODIFIED SHORT INTERFERING NUCLEIC ACID MOLECULES THAT MEDIATE RNA INTERFERENCE | 2006 |
|
RU2418068C2 |
| TRIALKYL CATIONIC LIPIDS AND METHODS FOR USING THEM | 2013 |
|
RU2718053C2 |
| TREATMENT OF CNS DISORDERS | 2007 |
|
RU2426544C2 |
| SMALL RNA MOLECULES, MEDIATING RNA INTERFERENCE | 2007 |
|
RU2470073C2 |
| SMALL RNA MOLECULES MEDIATING RNA INTERFERENCE | 2001 |
|
RU2322500C2 |
| PREVENTIVE OR THERAPEUTIC AGENT FOR TREATING FIBROSIS | 2011 |
|
RU2583290C2 |
| RNAI AGENTS, COMPOSITIONS AND METHODS FOR USE THEREOF FOR TREATING TRANSTHYRETIN (TTR) ASSOCIATED DISEASES | 2012 |
|
RU2678807C2 |
| TREATING DISEASES ASSOCIATED WITH VASCULAR ENDOTHELIAL GROWTH FACTOR (VEGF) BY SUPPRESSION OF NATURAL ANTISENSE VEGF TRANSCRIPT | 2009 |
|
RU2569182C2 |
