FIELD: biotechnology.
SUBSTANCE: described is a method for increasing the expression of transglutaminase 1 in the skin epidermis, comprising administering a recombinant adeno-associated virus based on a genetic construct subcutaneously, coding nucleotide sequence of transglutaminase 1 gene, where the genetic construct coding the nucleotide sequence of transglutaminase 1 gene comprises left and right terminal repeats (ITR), a promoter region comprising an enhancer and a promoter, a β-globin intron, Kozak sequence, an EcoRI restriction site, a codon-optimized sequence (coTGM1), a human growth hormone polyadenylation sequence (poly-A), replication sites, promoter and ampicillin resistance site sequence as shown in figure 1.
EFFECT: invention extends the range of methods for increasing the expression of transglutaminase 1 in the skin epidermis with a low immune response to the transgenic product.
1 cl, 8 dwg, 3 ex
| Title | Year | Author | Number |
|---|---|---|---|
| METHOD FOR THERAPY FOR TAY-SACHS DISEASE AND SANDHOFF DISEASE USING GENETICALLY MODIFIED HUMAN MESENCHYMAL STEM CELLS WITH OVEREXPRESSION OF β-HEXOSAMINIDASE A | 2020 |
|
RU2748383C1 |
| DRUG FOR THE TREATMENT OF METACHROMATIC LEUKODYSTROPHY AND A METHOD FOR ITS TREATMENT | 2021 |
|
RU2769577C1 |
| RECOMBINANT GENETIC CONSTRUCT, ADENO-ASSOCIATED VIRUS FOR THERAPY OF METACHROMATIC LEUKODYSTROPHY | 2023 |
|
RU2821568C1 |
| MINI-PROTEIN USH2A, NUCLEIC ACID CODING MINI-PROTEIN USH2A, AND EXPRESSION VECTOR FOR GENE THERAPY CONTAINING SAME | 2023 |
|
RU2822884C1 |
| CODON-OPTIMISED HAIPL1-ENCODING NUCLEOTIDE SEQUENCE AND EXPRESSION VECTOR CONTAINING SAID SEQUENCE | 2021 |
|
RU2785621C1 |
| MITOCHONDRIAL LOCALIZATION PEPTIDE, NUCLEIC ACID FOR ALLOTOPIC EXPRESSION OF MT-ND4 GENE CONTAINING ITS EXPRESSION VECTOR AND ITS USE | 2023 |
|
RU2817420C1 |
| VECTORS AND SEQUENCES FOR TREATING DISEASES | 2011 |
|
RU2588667C2 |
| NUCLEIC ACID FOR ALLOTOPIC EXPRESSION OF MT-ND4 GENE | 2023 |
|
RU2809065C1 |
| USING VECTOR FOR REDUCING IMMUNE RESPONSE IN VIRAL DELIVERY OF AIPL1 | 2023 |
|
RU2818342C1 |
| METHOD OF MYODYSTROPHY CORRECTION USING ADENO-ASSOCIATED VIRAL VECTOR IN EXPERIMENT | 2022 |
|
RU2821544C1 |
