FIELD: medicine.
SUBSTANCE: invention refers to medicine, particularly to experimental pharmacology and genetic technologies. Described method involves using male dysferlin-deficient mice B6.A/J-Dysfprmd and single introduction of two-vector preparation based on adeno-associated virus in tail vein with assessment of muscle function 30 days after injection of preparation. Correction of myodystrophy is carried out on the basis of adeno-associated virus of serotype 9 carrying a codon-optimized cDNA of the dysferlin gene under control of a muscle-specific promoter in a combination with a chimeric intron for enhancing the expression of dysferlin in volume of 100 mcl, confirmed by the results of the Grip strength test.
EFFECT: invention can be used for treating muscular dystrophy.
1 cl, 5 tbl, 1 ex
| Title | Year | Author | Number |
|---|---|---|---|
| METHOD FOR CORRECTING MUSCULAR DYSTROPHY USING PLASMID DNA IN DYSFERLIN-DEFICIENT MICE | 2022 |
|
RU2805357C1 |
| NUCLEIC ACID FOR ALLOTOPIC EXPRESSION OF MT-ND4 GENE | 2023 |
|
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| MINI-PROTEIN USH2A, NUCLEIC ACID CODING MINI-PROTEIN USH2A, AND EXPRESSION VECTOR FOR GENE THERAPY CONTAINING SAME | 2023 |
|
RU2822884C1 |
| ISOLATED MODIFIED CAPSID PROTEIN VPI OF ADENO-ASSOCIATED VIRUS SEROTYPE 5 (AAV5), CAPSID AND VECTOR BASED THEREON | 2021 |
|
RU2820088C1 |
| MITOCHONDRIAL LOCALIZATION PEPTIDE, NUCLEIC ACID FOR ALLOTOPIC EXPRESSION OF MT-ND4 GENE CONTAINING ITS EXPRESSION VECTOR AND ITS USE | 2023 |
|
RU2817420C1 |
| USING VECTOR FOR REDUCING IMMUNE RESPONSE IN VIRAL DELIVERY OF AIPL1 | 2023 |
|
RU2818342C1 |
| 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 |
| 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 |
| COMBINATION OF VECTORS FOR THERAPY OF CONGENITAL DYSFUNCTION OF ADRENAL CORTEX | 2023 |
|
RU2812469C1 |
| EXPRESSION VECTORS BASED ON ADENO-ASSOCIATED VIRUS | 2023 |
|
RU2812468C1 |
