Since all proof-of concept studies were conducted in mouse models, an important caveat is that the clinical efficacy of TN-401 and other PKP2 gene therapy programs or proof-of-concept studies62–64 (https://rocketpharma.com/aav-presentations-and-posters/; https://www.lexeotx.com/post/lexeo-therapeutics-announces-data-presentations-at-the-26th-american-society-of-gene-cell-therapy-asgct-annual-meeting/) remain to be evaluated in patients with ARVC caused by PKP2 mutations. The gene discussed is PKP2; the disease is arrhythmogenic right ventricular cardiomyopathy.