Researchers successfully disabled a gene in human patients by treating them with CRISPR gene editing technology, clearing patients’ blood of a toxic protein for some patients by as much as 93 percent up to six months after the initial treatment. The researchers detailed the findings in a press release, a phase 1 clinical trial update, and data slides on Monday (February 28).
“It is quite remarkable that this first [intravenous] CRISPR-based gene-editing effort has been so successful,” gene therapy researcher Terence Flotte of the University of Massachusetts Medical School, who was not involved with the study, tells Science. “This demonstrates great potential for the power of this platform clinically.”
The 15 patients, who are enrolled in a clinical trial conducted by the pharmaceutical companies Intellia Therapeutics and Regeneron Pharmaceuticals, have an inherited gene mutation called transthyretin (TTR) amyloidosis, a progressive neurological disease that causes numbness, nerve pain, and heart failure. ...
