CRISPR Strikes Gold with New Technique to Repair DNA

October 30, 2017

Gene editing with nanoparticles could lead to new treatments for genetic diseases

CRISPR, a technique that allows for the direct modification of nuclear DNA, has been making waves this year with results like the correction of a disease-causing mutation in human embryos.

Scientists at UC Berkeley recently developed a novel CRISPR delivery system that, unlike past systems, doesn’t rely on viruses. Instead, it uses gold nanoparticles to bind all the CRISPR components together into a compound called CRISPR-Gold. When injected, the CRISPR-Gold targets specific mutations, like the mutation that causes Duchenne muscular dystrophy. A single injection into the muscle tissue of mice with Duchenne doubled their strength and agility.

“CRISPR-Gold and, more broadly, CRISPR-nanoparticles open a new way for safer, accurately controlled delivery of gene-editing tools,” said UC Berkeley Professor Irina Conboy. “Ultimately, these techniques could be developed into a new medicine for Duchenne muscular dystrophy and a number of other genetic diseases.”