Silencing a Gene Lowers Cholesterol in Mice

A study published in Nature shows that targeted epigenetic silencing of the PCSK9 gene in mice reduced cholesterol levels that lasted for up to 330 days. The findings of the study provide significant insights into the development of treatments for diseases caused by high cholesterol levels.

Epigenome editing is an emerging technology that silences genes without altering the primary DNA sequence. According to Henriette O'Geen, an epigeneticist at the University of California, Davis, “This can alter the expression of genes that are involved in disease — and potentially provide a cure — without changing DNA.”

PCSK9, a protein involved in the degradation of LDL receptors in the liver, has long been a target for lowering cholesterol in both pharmaceutical and gene editing studies. Researchers from San Raffaele Scientific Institute in Milan, Italy, developed a system using zinc-finger proteins designed to target the PCSK9 gene. This breakthrough offers a one-shot approach that opens up new possibilities for therapeutics.

For more information, read the article from Nature.

See https://www.isaaa.org/kc/cropbiotechupdate/article/default.asp?ID=20696

Figure: A zinc-finger protein (blue and magenta; artificially coloured) in complex with DNA (yellow and green).Credit: Laguna Design/SPL.