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A single gene edit

By July 10, 2022January 11th, 2023No Comments

A single gene edit to reduce cholesterol…

The Verve trial aims to edit cells directly in the body. The team has encased the base-editing components — messenger RNA encoding the enzyme needed to alter DNA, and an extra snippet of RNA that will direct the enzymes to the correct location — in lipid nanoparticles, similar to those used in the formulation of mRNA COVID-19 vaccines. The nanoparticles will be concentrated in the liver, a key site ofPCSK9 is production.

And where this is going…

Other base-editing therapies are being developed to treat conditions such as leukaemia; a rare metabolic condition called glycogen storage disease; and Stargardt’s disease, which can cause blindness. And other CRISPR-derived approaches are being readied for their own foray into the clinic. Alternative Cas enzymes have been discovered that can edit RNA rather than DNA. Schwank says that his lab has mostly moved on from base editing, to a technique called prime editing, which offers more precision: “It’s all moving fast.”

Full article in Nature

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