This article appeared in the January/February 2022 issue of Discover magazine." Become a subscriber for unlimited access to our archive.
The CRISPR-Cas9 gene-editing tool once again ventured into uncharted territory this past year — in more ways than one.
Most notably, the gene editor — which functions like molecular scissors to snip apart DNA and insert, delete or modify genes — went to work inside human patients. In a landmark trial, a group of scientists from the U.K. and New Zealand showed that CRISPR can be used to successfully edit genes in the human body. Their work involved six patients with a devastating genetic disease called transthyretin amyloidosis, and was published in the New England Journal of Medicine in June. The hereditary form of the disease stems from mutations to the TTR gene, which produces the protein transthyretin. Patients experience progressively debilitating symptoms as misshapen protein clumps called amyloids build ...
