UNC Logo Duchenne muscular dystrophy is a fatal disease that exists for 1 in 3500 newborn boys where patients usually lose their capacity to walk and breathe. A study conducted by University of North Carolina at Chapel Hill scientists, has revealed a molecular method that seemingly moves one step closer to becoming a treatment for Duchenne muscular dystrophy.

This new therapy involves strips of genetic code called antisense oligonucleotides that strive to resurrect the working of an impaired dystrophin gene. Scientists have illustrated in the Ib and IIa trial of the strategy that corrects the growth of the crucial muscle protein which patients with progressive neuromuscular condition lack.

“When I first tried my approach in a test tube some twenty years ago, a reviewer of my manuscript commented that it was ‘molecular gymnastics that will never amount to anything. Now we have evidence that it works, and in an illness that has no other good therapeutic options,” remarked study co-author Ryszard Kole, PhD, a professor of pharmacology who has taken a leave of absence from UNC to develop the technology with AVI Biopharma, in Bellevue, Washington.

As part of the analysis, the scientists gauged an avenue to transform the lethal disease to a condition that permits survival by utilizing antisense oligonucleotides. These are strings of genetic lettering that get glued to and unravel the sections of code. The latter lead to the cell’s ‘splicing’ mechanism that contributes to the cutting and pasting of the guidelines collectively. They also skipped some paragraphs so that the frame of reading reverted to the right track and the remaining forms of dystrophin protein ought to be created as per the guide.

To affirm this procedure, 19 Duchenne muscular dystrophy patients were administered the therapy intravenously up to 12 weeks. It came to fore that the medicine was well accepted and seemingly raised the proportions of dystrophin protein based on dose-reliability. The 3 responders who were the best apparently showed a rise from 2% to 18%, from 0.9% to 17% and from 0% to 7.7% of normal muscle, correspondingly. The scientists have plans of expanding the study by increasing the dose and the time period of the process that will present its clinical effect. Future analyses may administer the drug subcutaneously or in tablet form.

The study is published in the July 25 issue of the journal The Lancet.