Gold Nanoparticles Ship With Efficiency CRISPR Cargo

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. The first of these problems has vexed the field since the gene-editing technique was discovered. Scientists need to deliver CRISPR into the right spot in a cell. That is proving tricky enough. DNA represents the body’s crown jewels, and CRISPR must sneak past all sorts of security systems to gain access.

And then CRISPR must go global. Gene editing could benefit millions of people worldwide. But as the treatment process stands right now, the vast majority won’t. That process depends almost entirely on highly engineered viruses made in high-tech, multimillion-dollar facilities.
The researchers think their golden nanoparticles can solve both problems. As efficient couriers, they could reduce the need for engineered viruses and specialized research centers. And that could help make these emerging, high-tech treatments accessible and affordable, said senior scientist Dr. Jennifer Adair of Fred Hutch.

Gene therapy has a lot of potential across many diseases, but the process we have right now is just not feasible in every place in the world,” Adair said. “We want to end up delivering gene therapy in a syringe. This gold nanoparticle represents the first possibility we have to do that for blood stem cells.”

Source: https://www.fredhutch.org/