antisense oligonucleotides
Antisense oligonucleotides are short, synthetic strands of DNA or RNA designed to bind to specific messenger RNA (mRNA) molecules. By attaching to these mRNA strands, they can inhibit the production of proteins associated with certain diseases, effectively reducing or altering their expression.
These molecules are used in various therapeutic applications, particularly in treating genetic disorders and some types of cancer. By targeting the mRNA that codes for harmful proteins, antisense oligonucleotides can help manage conditions like Duchenne muscular dystrophy and spinal muscular atrophy, offering new avenues for treatment.