Forget UPS and FedEx: Tiny golden delivery trucks created at Fred Hutchinson Cancer Research Center can ship CRISPR into human blood stem cells, offering a potential way to treat diseases like HIV and sickle cell anemia.
And the researchers behind those trucks have even bigger distribution dreams.
Gene therapy — the editing of our DNA to treat disease — is a clinical reality today, but only in a handful of rich countries. Fred Hutch scientists think their new CRISPR courier could help deliver gene therapy to patients around the world.
A new paper published in Nature Materials describes how the scientists loaded CRISPR onto spherical gold nanoparticles. These tiny shuttles then deposited the gene-editing tool into blood stem cells donated by healthy individuals and isolated in test tubes, where CRISPR altered genes related to HIV and certain blood disorders.
It is the first time that nanoparticles have successfully ferried CRISPR into blood stem cells to edit DNA, the researchers said. And it’s a promising step toward addressing CRISPR’s critical delivery problems.
