Scientists at the Institute of Chemical Biology and Fundamental Medicine (ICBFM) of the Siberian Branch of the Russian Academy of Sciences have successfully modified DNA fragments for the treatment of spinal muscular atrophy (SMA). This innovative modification of the sugar-phosphate backbone provided high stability of nucleic acids in biological fluids. The result was a significant reduction in the required dosage of the drug and a decrease in the risks associated with injections into the cerebrospinal fluid. The information was provided by the Acting Director of ICBFM SB RAS, Vladimir Koval.
The therapeutic oligonucleotides synthesized by the scientists—short DNA fragments—proved to be significantly more stable in biological environments compared to natural DNA. This substantial improvement in stability will reduce the number of injections of the costly drug, ultimately lowering expenses.
SMA is a severe genetic disorder, and in Russia, doctors identified it in 55 newborns through expanded neonatal screening in August 2023. Currently, there are two registered drugs for treating this condition: “Spinraza” and “Zolgensma.” However, both drugs require frequent injections, and their cost is quite high.
The development by ICBFM scientists could significantly reduce the costs of SMA therapy by providing more stable and affordable oligonucleotide drugs. Additionally, this technology may serve as the basis for developing therapies for other genetic diseases, such as muscular dystrophies, age-related muscular degeneration, and more.
Photo: msk.medicalgenomics