TTR and Familial transthyretin-related amyloidosis: Promising current clinical trials for direct in vivo Cas9 gene editing strategies are based on lipid nanoparticle (LNP)-mediated Cas9 delivery for the treatment of hereditary angioedema and for transthyretin amyloidosis, by disrupting liver expression of KLKB1 or TTR, respectively40,41.