Antisense Oligonucleotides (ASOs) can be used to block or stimulate mRNA resulting in blocking or promoting the target protein.
What is the difference between ASO and siRNA? ASOs have one strand while siRNAs have two. Can help cure various genetic diseases. Key challenge is drug delivery.
ASO drugs end with '-rsen'. Antiviral ASOs end with -'virsen'.
Can be used to treat an individual patient with a unique disease thus raising regulatory/ethical concerns.
Milasen became the first drug approved for a single person named Mila.
 Wikipedia. Antisense Therapy. https://en.wikipedia.org/wiki/Antisense_therapy
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 New Eng Journal of Med. Treating Disease at the RNA Level with Oligonucleotides. 01-2019
 Biogen. Understanding Antisense Oligonucleotides. 12-2020
 Biogen. Antisense oligonucleotides (ASOs). 05-2019
 Fox31 Denver. Colorado girl is first person to receive FDA-approved drug created for one patient. 12-2019