Another sublime example is provided by Gillmore et al., who conducted a preclinical investigation (Intellia Therapeutics, 2020) which showcases the significant efficacy and safety of NTLA-2001, a novel in vivo CRISPR/Cas9-mediated gene therapy for the treatment of Transthyretin (TTR) Amyloidosis, also called ATTR Amyloidosis (Gillmore et al., 2021). The gene discussed is TTR; the disease is amyloidosis.