We note that, there still remain challenges in the research that need to be urgently studied, including: 1) the discovery of compounds to reverse the association state of TTR from amyloidal state to native folding state; 2) the misfolding structures of TTR amyloidal oligomers and the structure-based drug design to specifically target the amyloidal intermediates of TTR; 3) the therapeutic approaches to promote cardiac repair and preserve cardiac function against the dysfunction caused by TTR amyloidosis. The gene discussed is TTR; the disease is amyloidosis.