FIELD: molecular biology; virology; biomedicine.
SUBSTANCE: invention relates to cell technologies. Described is a method for maintaining static endurance in dysferlin-deficient mice in experiment, involving use of male dysferlin-deficient mice B6.A/J-Dysfprmd and administering a two-vector adeno-associated virus preparation. A preparation based on adeno-associated virus of serotype 9 is administered intramuscularly (i. m.), carrying a codon-optimized cDNA of the dysferlin gene under the control of a muscle-specific promoter in combination with a chimeric intron to enhance dysferlin expression in dose of 5*1012 units virus with the DYSF gene in amount of 50 mcl in the following muscles of both hind limbs: m. vastus lateralis, m. tibialis anterior, m. gastrocnemius lateralis per animal in total 300 mcl of mice B6.A/J-Dysfprmd at age of 6 months with repeated administration of preparation 1 month after the first administration with assessment of physical performance 3 months after the first administration of the preparation, which is confirmed by the results of the test “Animal retention on a slippery vertical rod”.
EFFECT: invention can be used to preserve static endurance.
1 cl, 4 tbl, 1 ex
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Authors
Dates
2024-11-05—Published
2023-12-26—Filed