Preliminary results from an ongoing trial by Intellia Therapeutics show that a CRISPR-Cas9-based drug can be delivered into the body to target the liver and reduce expression of the gene that causes transthyretin amyloidosis (ATTR).1 This is the first clinical trial demonstrating successful in vivo gene editing; the results suggest that it may be possible to safely edit the genomes of cells in the body.
“For in vivo delivery, the goal is that you can administer CRISPR as a medicine to the patient,” said Laura Sepp-Lorenzino, chief scientific officer of Intellia Therapeutics.
ATTR is characterized by a misfolded version of the transthyretin (TTR) protein that builds up in the heart, nervous system, and kidneys. Patients generally experience pain, weakness, and the inability to control basic body functions. “It's a truly dreadful condition,” said Julian Gilmore, a physician-scientist at the National Amyloidosis Centre at University College London, who led the trial.
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