METHOD OF MYODYSTROPHY CORRECTION USING ADENO-ASSOCIATED VIRAL VECTOR IN EXPERIMENT Russian patent published in 2024 - IPC C12N15/113 C12N15/63 C12N15/864 A61K48/00 

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.

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