Recent studies and clinical trials show that NTLA-2001 successfully reduced TTR proteins, considered responsible for ATTR (transthyretin amyloidosis). The NTLA-2001 was used in a clinical study, and this gene-editing technology proved to be a success. Intellia Therapeutics and Regeneron Pharmaceuticals’ conducted the study. They stated that the Phase 1 study was meant to see if NTLA-2001 was effective in patients suffering from hereditary transthyretin amyloidosis with polyneuropathy.
What is NTLA-2001?
According to the publication, the NTLA-2001 is a CRISPR therapy administered to patients via intravenous infusion. Systematical administration via intravenous infusion, the NTLA-2001 proved it could edit genes inside human tissues. Therefore the administration was meant for targeted tissues so that it would inactivate TTR genes inside liver cells. Once the TTR gene is inactivated, then there is no more production of the TTR protein. This means the patient does not develop complications of ATTR.
CRISPR-Cas9 in vivo gene editing might be the breakthrough everyone was looking for
The study mentions that using the in vivo gene-editing technology, those suffering from the dangerous ATTR disease could receive better treatment. There have been preclinical in vitro and in vivo studies to reach this conclusion, and there are ongoing clinical trials. The findings show that the CRISPR-Cas9 therapy is effective, and it can edit almost all genes inside liver cells that causes the mutation. The therapy would be vital as it could help edit several mutations responsible for more than 6,000 diseases. Researchers are still investigating which is the best way to administer the CRISPR therapy. So far in the existing clinical trials, the therapy has been injected into the bloodstream, into the patients’ eyes and via intravenous infusion. After several clinical trials are ready, more news will see how we can provide better treatment for diseases related to genetic mutations.