If the first CRISPR decade was marked by a mad dash to evolve, engineer, and mine the bacterial universe for more and better genome-editing enzymes, so far the second seems to be all about three words: “Delivery. Delivery. Delivery.”
Gene-editing technologies hold huge promise for treating hundreds, even thousands of inherited diseases. But many such disorders involve tissues researchers still can’t reliably deliver CRISPR systems to — like neurons, muscles, and blood stem cells. Developing new methods for smuggling CRISPR systems past the biological barriers that human cells have erected to safeguard the DNA coiled inside their watery guts is now the focus of an intensifying race among top labs and deep-pocketed investors.
Turning powerful gene-editing technologies into broadly useful therapies will require something at once more simple and far more complicated — in vivo editing approaches that use a single shot or infusion of CRISPR, packaged in such a way that it won’t fall apart in the bloodstream or set off immune alarm bells and can cross a cell membrane. In the pipeline are treatments that attempt to accomplish this by borrowing stealthy technologies from other fields, like gene therapy’s viral vector workhorse, AAV, or the lipid nanoparticles that coat RNA-interfering drugs and mRNA vaccines.
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