Russian Scientists Revolutionize Inherited Eye Disease Treatment
Russian scientists at Sirius University of Science and Technology have made a significant breakthrough in treating inherited eye diseases. The new method, developed by the team, could potentially preserve sight in patients with severe retinopathies and pave the way for domestic drugs to treat these conditions.
The innovative technique involves 'assembling' large therapeutic proteins directly inside cells. This approach bypasses delivery issues faced by traditional gene therapy methods. The team has successfully tested the method in kidney cells and retina cells, with a prototype drug for Stargardt disease showing promising results in animal trials.
The technology utilises a natural protein splicing mechanism, achieving an assembly efficiency of 70-80%. If further clinical trials are successful, Russia could have its own platform for developing advanced genetic drugs. This includes treating diseases such as cystic fibrosis, hemophilia A, hereditary deafness, and Duchenne muscular dystrophy, in addition to various retinopathies.
The Sirius team is currently conducting tests for biological safety and preparing for future clinical trials. While the specialists involved remain unnamed, their work offers new hope for patients and their families. Successful implementation of this method could revolutionise the treatment of inherited diseases, both in Russia and globally.
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