 Intellia shows CRISPR gene editing in humans has arrived. Intellia has released clinical data from NTLA-2001. Transthyratin amyloid dosis is the buildup of misfolded transthyratin, a protein generated by the liver. This protein is normally partially responsible for the transport of thyroid hormones, but misfolded versions form protein clumps and can lead to neuropathy. NTLA-2001 is a gene therapy that uses the well-known CRISPR-Cas9 gene editing system to knock out the TTR gene, preventing the liver from making transthyratin. People who received a lower dose of 0.1 mg per kg had their transthyratin halved, while people who received a higher dose of 0.3 mg per kg lost a full 5-6th of their transthyratin. This could have implications for aging because another version of transthyratin amyloid dosis exists, senile systemic amyloid dosis which predominantly affects very old people. Subscribe to our X-Ten YouTube channel for more videos.