The gene editor CRISPR has taken the whole world by storm, which has been widely used in the fields of human biology, agriculture and microbiology due to its accuracy, low cost and high efficiency. In term of medicine, Intellia and Regeneron have recently obtained positive mid-term data in clinical trials using CRISPR for the treatment of transthyroxine amyloidosis (ATTR). This breakthrough indicates that CRISPR gene editing has been expected to combat diseases that were once considered to be invincible.
Hereditary thyroxine transporter protein (ATTRV) amyloidosis is a genetic degenerative disease caused by mutations in the gene for TTR, which causes the liver cells to churn out misfolded forms of the transthyretin and accumulate in the nerves and the heart. The main manifestations of the disease are polyneuropathy (ATTRv-pn) or cardiomyopathy (ATTRv-cm). The CRISPR clinical trial aims to deactivate the mutated gene. Clinical data showed that the level of TTR protein in patients treated with CRISPR injection decreased sharply after several weeks, and there was almost no short-term side effect. The mid-term data of this trial was reported at the annual meeting of the Peripheral Nerve Society (PNS) in 2021 and published in The New England Journal of Medicine. Current clinical trials involve only six patients, and the team still needs long-term studies to examine possible negative effects.
Livers are better adapted to CRISPR therapy than other body organs or tissues because it sops up foreign particles. The success of this study paves the way for CRISPR treatment of other liver diseases. Based on the success of CRISPR test, there are reasons to believe that we are embarking on a new era of medicine.