Hitting landmark milestone, Intellia shows CRISPR can edit genes directly in patients
In a landmark study, researchers successfully used CRISPR to directly edit DNA in humans, a milestone that could pave the way for treatments for scores of serious genetic and non-genetic diseases.
Intellia, a biotech co-founded by 2020 Nobel laureate Jennifer Doudna, used CRISPR/Cas9 to cut a gene out of the liver cells of patients with ATTR amyloidosis, a deadly disease where a misshapen protein called TTR builds up and damages organs throughout the body. Patients who received a high dose of the therapy saw their protein levels fall between 80% and 96%, indicating the therapy permanently cut the genome where desired.
To read Endpoints News become a free subscriber
Unlock this article instantly, along with access to limited free monthly articles and our suite of newsletters