Personalized antisense oligonucleotides (ASOs) may accelerate studies on treating Duchenne muscular dystrophy (DMD).
Every year, tens of thousands of babies are born with enigmatic illnesses brought on by genetic abnormalities that are so uncommon that no treatment has yet been attempted. Researchers have now discovered a method to expedite the laboratory work required to test customized medications for some of these ill kids by using tiny blobs of tissue produced from patients’ blood cells.
When used to treat rare genetic diseases, personalized antisense oligonucleotides (ASOs) have shown promising results. The capacity to identify individuals with uncommon diseases who have pathogenic genetic variations susceptible to this therapeutic approach is likely to improve as clinical sequencing technology continue to progress.