Even more recently, the safety and pharmacodynamic effects of single escalating doses of NTLA-2001, a lipid nanoparticle encapsulating messenger RNA for Cas9 protein and a single guide RNA targeting the TTR gene to accomplish in vivo gene editing, have been investigated in six patients with hereditary ATTR amyloidosis with polyneuropathy, within an ongoing phase 1 clinical study [147]. Here, TTR is linked to polyneuropathy.