TTR and polyneuropathy: This proof-of-concept study showed remarkable long-term effectiveness (more than 12 months), achieving a >97% reduction in serum mouse TTR levels with a single administration.186 Subsequent clinical trials in patients with ATTRv polyneuropathy showed a dose-dependent, durable TTR reduction with minimal side effects, solidifying in vivo CRISPR/Cas9-mediated genome editing as a promising therapeutic avenue for ATTRv.187 Also, studies using mAbs that specifically targeted disease-associated WT and variant ATTR aggregates in normal mice have yielded encouraging results.