CRISPR ‘Cousin’ Put to the Test in Landmark Heart-Disease Trial

July 15, 2022

(Nature) – It’s test time for CRISPR’s cousin. A clinical trial that recently treated its first participant will test whether base-editing — a genome-editing method related to the CRISPR–Cas9 system — can safely be used to make precise, single-letter changes to a DNA sequence in a cholesterol-regulating gene without breaking both strands of DNA first, as CRISPR–Cas9 would do. This study will be followed by another base-editing trial, slated to treat its first participant later this year, that will aim to treat sickle-cell disease, a genetic blood disorder. (Read More)