The biotech company Verve Therapeutics launched the study with the aim of using base editing to treat a genetic condition that causes high cholesterol and increases a person's risk of developing cardiovascular disease.
This month marks ten years since CRISPR-Cas9 was repurposed as a gene editing system, so we’re looking back at what has been accomplished in a decade of CRISPR editing.
The approach, called GRAND, uses a second guide RNA to write complementary strands of DNA in targeted genomic locations, resulting in efficient insertions that can be hundreds of base pairs long.
A CRISPR-based system that reverses epigenetic changes caused by adolescent binge drinking reduces adult addiction-like behaviors in rats, a study finds, suggesting that an epigenomic approach could someday help treat people with alcohol use disorder.
A first-of-its-kind gene therapy dramatically reduced misfolded protein levels in some clinical trial participants for up to six months and reduced levels in all participants for up to a year.