First Gene-Editing Treatment Injected Into the Blood Reduces Toxic Protein for up to 1 Year

March 1, 2022

(Science) – The first team to disable a disease gene directly in a person through an infusion of the genome editor CRISPR reported yesterday that levels of the toxic protein made by the gene dropped as much as 93% for up to 1 year. The researchers hope the long-term reduction means patients in the clinical trial will soon show clear improvement in their nerve symptoms and that these benefits will persist for an extended period. (Read More)