An ASO is a short synthetic single strand of DNA that targets a specific region of a gene that produces a faulty protein known to cause disease. It can reduce production of that faulty protein or stop it from being expressed completely, or restore function to that protein.
Antisense oligonucleotide
Kiran Goyal
I work as a Research Officer within the MND Association to help organise the International Symposium and communicating the latest updates of MND research across our social media platforms. I graduated with a master's degree in Neuroscience from Cardiff University in 2023. I have previously supported the awareness of Fragile X syndrome within the UK.