FIELD: biotechnology.
SUBSTANCE: microRNA specific for PLP1 gene, containing a pair of nucleotide sequences, consisting of an antisense sequence and a sense sequence, a vector capable of suppressing expression of PLP1 gene, a pharmaceutical composition for the treatment of a disease related to PLP1 gene anomaly, and an inhibitor of expression of PLP1 gene are presented. A method for the treatment of a disease related to PLP1 gene anomaly and a method for suppression of expression of PLP1 gene are also presented.
EFFECT: invention allows for use it for the treatment of a disease related to PLP1 gene anomaly, for example, such as Pelizaeus-Merzbacher disease.
19 cl, 13 dwg, 3 tbl, 10 ex
| Title | Year | Author | Number |
|---|---|---|---|
| COMPOSITIONS AND METHODS FOR TREATING HAEMOGLOBINOPATHIES | 2015 |
|
RU2707540C2 |
| PROMOTER COMPOSITIONS | 2014 |
|
RU2742435C2 |
| THERAPEUTIC COMPOUNDS FOR TREATING HUNTINGTON'S DISEASE | 2015 |
|
RU2711147C2 |
| MODIFIED ADENO-ASSOCIATED VIRUS VECTOR COMPOSITIONS | 2013 |
|
RU2679843C2 |
| DOUBLE-STRANDED RNA CAPABLE OF REDUCING EXPRESSION OF MUTANT ALLELE C.607G>A OF HUMAN GNAO1 GENE | 2022 |
|
RU2816137C1 |
| AAV-BASED HUNTINGTON'S DISEASE TREATMENT | 2017 |
|
RU2749971C2 |
| SEQUENCE DISPOSED UPSTREAM FROM CARP GENE, VECTOR CONTAINING THE SAME AND USES THEREOF | 2001 |
|
RU2283865C2 |
| VECTORS AND SEQUENCES FOR TREATING DISEASES | 2011 |
|
RU2588667C2 |
| CLOSED-ENDED DNA VECTORS OBTAINED BY CELL-FREE SYNTHESIS, AND METHOD OF PRODUCING CEDNA VECTORS | 2019 |
|
RU2820586C2 |
| RNAi AGENT OPTION | 2018 |
|
RU2789647C2 |
