Antisense Oligonucleotides and colony Stimulating Factors

Antisense oligonucleotides are short, single-stranded DNA molecules that interact with messenger RNA to prevent translation of a target gene. In addition to its unlimited potential for therapeutic applications, antisense technology has been widely used in agricultural bioengineering to improve crop trait, known as gene silencing. Colony stimulating factors are glycoproteins that promote production of white blood cells (mainly granulocytes such as neutrophils), in response to infection. Administration of exogenous colony stimulating factors stimulates the stem cells in the bone marrow to produce more of the particular white blood cells.

Antisense oligopeptides (ASO) are named with the suffix of –sen in their nomenclature. Early approval includes Formivirsen (2002) and Mipovirsen (2013). Because of its great therapeutic potential, numerous ASOs are in current development inventory. In 2019, FDA granted accelerated approval to an antisense oligonucleotide (ASO) therapy, Golodirsen for a genetic neuromuscular diseases, Duchenne muscular dystrophy. The best known colony stimulating factors are Amgen’s Neupogen (filgrastim) and Neulasta (peglylated filgrastim), used to reduce chemotherapy-induced infection.