Gene therapy test launches pivotal year for precise genome-editing technique known as base editing.
Credit: Martin Steinthaler/GettyA clinical trial that recently treated its first participant will test whether base-editing — a genome-editing method related to the CRISPR–Cas9 system — can safely be used to make precise, single-letter changes to a DNA sequence in a cholesterol-regulating gene without breaking both strands of DNA first, as CRISPR–Cas9 would do.
Both tests are expected to report results in 2023, and further base-editing treatments are working their way through the pipeline towards clinical trials. “It’s very exciting that the first clinical trials are starting, with CRISPR–Cas9 and now also with base editing,” says Gerald Schwank, who studies the use of genome editing to treat diseases at the University of Zurich, Switzerland. “We’ve got a lot to learn.
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