» Tricyclo-DNA for Duchenne Action Duchenne: "Recently, researchers from Bern developed an active substance, which together with an international team they tested successfully. This promising therapy approach is to correct the defect in the production of dystrophin with short segments of a chemically modified DNA, so-called antisense oligonucleotides (AO). A significant curative effect with the tested active substances had not been achieved until now. That is because the corresponding active substances are not yet active enough and do not reach vital muscles such as the heart.
Experiments in mice show evidence that the agent leads to an improved dystrophin production in all muscles including the heart and the lungs and subsequently leads to an improved mobility and an increased life expectancy in mice.
Surprisingly it was also observed that the dystrophin production in the brain was corrected as well. It was thus shown for the first time that an oligonucleotide can pass the blood-brain barrier and become active there.
The next step of the clinical trial is to test tricyclo-DNA in humans. The trial is coordinated by the spin-off enterprise Synthena AG of the University of Bern founded in 2012. The enterprise manufactures tricyclo-DNA and advances the development of a medication for Duchenne patients.
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