A new form of therapy might stop or can even reverse a form of progression eye sight loss, which till now has caused blindness. This therapy is likely to be boon, a ray of hope for people suffering with spinocerebellar ataxia type 7. This therapy is expected to treat neurogentic diseases, with no or minimal side effects in comparison to other medications. In the recent issue of the Science Translational Medicine, a detailed report about the therapy is published.
Al La Spada, MD, Ph.D and a senior author to this study, with his colleagues have come up with a therapy form known as antisense oligonucleotides (ASOs). ASOs are single stranded pieces of DNA which are known to bind to specific RNA molecules. ASOs are likely to offer a promising results as they attack directly on the harmful proteins which are produced by various genetic disorders. In recent times, Spinraza therapy is used mostly for patients with spinal muscular atrophy. This therapy uses identical technique that of ASOs and have come out to be effective by stopping motor neuron loss.
In order to check the efficiency of the therapy, the team made ASOs that matched with the mouse’s RNA. After that, they injected the ASOs in the vitreous humor of one eye of SCA7 mice, while on the other eye, they dropped saline solution. After 4 and 6 weeks of treatment, it was found that eye having injected with ASOs had better results. It had better visual function in comparison to the other one.