Transthyretin amyloidosis is a monogenic hereditary disease resulting from the accumulation of abnormal transthyretin protein, leading to organ dysfunction and death.[170] As the liver is the primary site for transthyretin (TTR), a hepatocyte‐targeted LNP was developed to deliver Cas9‐sgRNA mRNA precisely into hepatocytes and alleviate TTR retention. This evidence concerns the gene TTR and Familial transthyretin-related amyloidosis.