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
Research Information Team
The MND Association’s vision is a world free from MND. Realising this vision means investing more in research, further developing partnerships with the research community, funding bodies and industry, while ensuring that advances in understanding and treating MND are communicated as quickly and effectively as possible.