FIELD: biotechnology; molecular biology; genetic engineering.
SUBSTANCE: nucleic acid is proposed for allotopic expression of MT-ND4 gene, as well as an expression vector containing the said nucleic acid, and its use for the treatment of Leber hereditary optic neuropathy. The nucleic acid contains the nucleotide sequence SEQ ID No. 1 and the sequence encoding the mitochondrial localization peptide of cytochrome c oxidase subunit 8. The inventions make it possible to reduce the level of reactive oxygen species in the cell by at least 25% and the level of mitochondrial calcium by at least 29%.
EFFECT: inventions can be used for gene replacement therapy for Leber hereditary optic neuropathy.
11 cl, 11 dwg, 2 tbl, 8 ex
| Title | Year | Author | Number |
|---|---|---|---|
| MITOCHONDRIAL LOCALIZATION PEPTIDE, NUCLEIC ACID FOR ALLOTOPIC EXPRESSION OF MT-ND4 GENE CONTAINING ITS EXPRESSION VECTOR AND ITS USE | 2023 |
|
RU2817420C1 |
| MINI-PROTEIN USH2A, NUCLEIC ACID CODING MINI-PROTEIN USH2A, AND EXPRESSION VECTOR FOR GENE THERAPY CONTAINING SAME | 2023 |
|
RU2822884C1 |
| NUCLEIC ACID DESIGNED TO REDUCE MAMMALIAN BODY WEIGHT, EXPRESSION VECTOR FOR EXPRESSION IN MAMMALIAN CELLS, METHOD FOR ITS DELIVERY AND METHOD FOR REDUCING MAMMALIAN BODY WEIGHT | 2023 |
|
RU2810191C1 |
| USING VECTOR FOR REDUCING IMMUNE RESPONSE IN VIRAL DELIVERY OF AIPL1 | 2023 |
|
RU2818342C1 |
| CODON-OPTIMISED HAIPL1-ENCODING NUCLEOTIDE SEQUENCE AND EXPRESSION VECTOR CONTAINING SAID SEQUENCE | 2021 |
|
RU2785621C1 |
| CODON-OPTIMIZED NUCLEIC ACID WHICH ENCODES SMN1 PROTEIN, AND USE THEREOF | 2020 |
|
RU2742837C1 |
| DOUBLE-STRANDED RNA CAPABLE OF REDUCING EXPRESSION OF MUTANT ALLELE C.607G>A OF HUMAN GNAO1 GENE | 2022 |
|
RU2816137C1 |
| EXPRESSION VECTOR BASED ON ADENO-ASSOCIATED VIRUS CARRYING RECOMBINANT ANTIBODY GENES AND ITS USE FOR PREVENTION OF DISEASES CAUSED BY INFLUENZA A VIRUS AND SARS-CoV-2 VIRUS | 2023 |
|
RU2817792C1 |
| METHOD FOR CORRECTION OF MITOCHONDRIAL DYSFUNCTION BY GENETIC CONSTRUCT | 2016 |
|
RU2642972C1 |
| REGULATORY SEQUENCE TO INCREASE GENE EXPRESSION | 2022 |
|
RU2804421C1 |
