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Siberian scientists have obtained a new compound for healing rare type of muscular dystrophy

Researchers from the Institute of Chemical Biology and Fundamental Medicine (ICBFM) of Siberian Division of Russian Academy of Science have synthesized a new substance which helps in healing laboratory mice with muscular dystrophy of Duchenne, a rare disease when a subject losses a moving ability. The achievement has been reported by the Chief of Laboratory of Chemistry of Nucleic Acids at ICBFM Dmitry Stetsenko.
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«Muscular dystrophy of Duchenne is caused by mutations in a gene of protein dystrophin — an essential component of healthy muscles. A possible approach for healing this disease is a correction of mutation using synthetic oligonucleotide (short fragment of DNA or RNA). Such preparation has been recently introduced in the USA. We have developed a new type of chemically modified oligonucleotides — phosphorylguanidines which have already shown good results comparable to those with foreign analogs when treating mice with muscular dystrophy of Duchenne,» - Stetsenko said.

Every year, about 20 thousand new cases of muscular dystrophy of Duchenne are fixed all over the world. In Russia, there live about 3.5 thousand subjects with such diagnosis.

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