TTR and Familial transthyretin-related amyloidosis: In humans, Gillmore et al successfully used a specialized LNP formulation in a clinical trial with an in vivo gene-editing approach to reduce the serum level of transthyretin (TTR) by targeting the TTR gene as a therapeutic approach to treat Transthyretin amyloidosis (ATTR) (Gillmore et al., 2021).