Volanesorsen: A novel treatment for high triglyceride levels

How do we treat genetic mutations? Previous mechanisms have focused on genome editing, however, recent studies are using antisense oligonucleotides! These short sequences bind to a complementary mRNA strand, inhibiting translation and formation of the encoded protein.
In 2013, Graham and colleagues set out to develop a treatment for one of the most common health problems - elevated triglycerides. Triglycerides are metabolised by hepatic lipase and lipoprotein lipase, however, apolipoprotein C-III (apoC-III) has been identified as hindering metabolism. Therefore, Graham and colleagues developed ISIS 308401 which aimed to promote triglyceride metabolism by targeting apoC-III mRNA.
After its success in healthy participants, it was approved (sort of) to treat familial chylomicronemia syndrome, a rare disorder causing hypertriglyceridemia due to lipoprotein lipase deficiency. However, many patients developed thrombocytopenia, so further modification is required.
Despite this, nucleic-acid therapies have a promising future to treat many conditions, giving patients a second chance at life.
Creator: Jacinta Murphy
References:
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