FIELD: biotechnology.
SUBSTANCE: invention refers to biotechnology and molecular biology. Disclosed is an adenoassociated virus (AAV)-based vector genome for inhibiting or suppressing superoxide dismutase 1 (SOD1) gene expression in a mammalian cell, as well as a corresponding particle, a method of inhibiting gene expression, a pharmaceutical composition, a method of treating amyotrophic lateral sclerosis and duplex siRNA.
EFFECT: inventions can be used in medicine to expand the range of agents for controlling amyotrophic lateral sclerosis.
23 cl, 12 dwg, 22 tbl, 18 ex
| Title | Year | Author | Number |
|---|---|---|---|
| THERAPEUTIC COMPOUNDS FOR TREATING HUNTINGTON'S DISEASE | 2015 |
|
RU2711147C2 |
| MODULATING POLYNUCLEOTIDES | 2015 |
|
RU2749882C2 |
| MODULATING POLYNUCLEOTIDES | 2015 |
|
RU2719192C2 |
| RNAi AGENT OPTION | 2018 |
|
RU2789647C2 |
| AADC POLYNUCLEOTIDES FOR TREATING PARKINSON'S DISEASE | 2015 |
|
RU2716991C2 |
| TREATMENT OF HBV INFECTION | 2011 |
|
RU2620966C2 |
| DOUBLE-STRANDED RNA CAPABLE OF REDUCING EXPRESSION OF MUTANT ALLELE C.607G>A OF HUMAN GNAO1 GENE | 2022 |
|
RU2816137C1 |
| COMPOSITIONS AND METHODS FOR INHIBITING TMPRSS6 GENE EXPRESSION | 2012 |
|
RU2702501C2 |
| LINES OF ENGINEERED PRODUCER CELLS AND METHODS FOR PRODUCTION AND USE THEREOF | 2020 |
|
RU2823068C2 |
| PEST CONTROL USING RNA MOLECULES | 2017 |
|
RU2801251C2 |
