antisense oligonucleotides

Antisense oligonucleotides (ASOs) are short strands of synthetic DNA or RNA designed to bind to specific messenger RNA (mRNA) in the body. By attaching to the mRNA, ASOs can block the production of harmful proteins associated with certain diseases. This innovative approach is being explored for treating various genetic disorders and conditions, such as muscular dystrophy and certain types of cancer. Essentially, ASOs serve as a targeted therapy, modifying how genes express themselves, thereby offering a new avenue for medical treatment based on genetic information.