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