Belgorod scientists tested a drug for rare forms of muscular dystrophy on mice
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- Belgorod scientists tested a drug for rare forms of muscular dystrophy on mice
A team of researchers from the Research Institute of Pharmacology of Living Systems at the National Research University BelSU has successfully investigated the effect of a drug based on adeno-associated viral vector intended for use in the therapy of dysferlinopathies, the press service of the Russian Ministry of Education and Science told Izvestia.
This is a group of rare (orphan) muscular dystrophies characterized by muscle weakness and atrophy of various muscle groups up to complete disability. The disease is rare, occurring in seven in 1 million newborns, and manifestations begin subtly, making diagnosis difficult.
"Our research group is involved in studying a drug based on an adeno-associated virus carrying a codon-optimized complementary DNA of the dysferlin gene under the control of a muscle tissue-specific linker. This allowed to enhance the expression of dysferlin, as well as to get rid of the disadvantages of the analog associated with contraindications to the use of the drug", - said about the uniqueness of the developed drug the head of the work, Professor of the Department of Pharmacology and Clinical Pharmacology of the Medical Institute of the National Research University "BelSU", Doctor of Medical Sciences Mikhail Korokin.
In the course of research on the effectiveness of the drug scientists observed a pronounced correction of physical performance in dysferlin-deficient mice.
According to the scientists of the National Research University "BelSU", the results obtained allow us to conclude that the investigated drug provides a pronounced expression of the dysferlin gene, and can be the basis of an effective means of physical performance correction for the therapy of dysferlinopathies.
Read more in Izvestia's exclusive story:
Power loading: a drug for rare forms of muscular dystrophy has been tested in the Russian Federation
