Matthew Porteus (Credit: Timothy Archibald for Stanford Children's)
Versant teams up with Stanford gene editing experts on a $45M next-gen play — marrying CRISPR and AAV to fix sickle cell
When a researchers talk about gene editing, they’re usually thinking about several steps. First you need to zero in on the defective gene; then, depending on the need, you’d want to knock out, replace or insert genetic material.
CRISPR/Cas9 technologies have transformed the field by making a breakthrough for the first problem. Inducing double-stranded DNA breaks, or achieving single-letter changes as base editing allows, have promising applications in multiple diseases that are starting to get tested in humans.
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