So let me get this out of the way upfront: I’m no scientist—and no, I don’t play one on television. I struggled with science in school, which is why I’m opening an article like this by apologizing for my lack of science…ness.

But while my scientific acumen will never put me in the running for a Nobel Prize, I can at least recognize and appreciate a significant breakthrough.

And a breakthrough in treating tyrosinemia type I is exactly what a group of researchers at the University of Massachusetts Medical School may have discovered.

Here’s the deal: Wen Xue, PhD, is an assistant professor of molecular medicine at UMass and part of a team studying something called the CRISPR/Cas9 gene editing system. The system is designed to help cut into DNA using a “molecular scalpel”; that’s the Cas9 part. Meanwhile, an RNA guide component helps direct the scalpel to the exact spot to cut into the DNA. By doing that, scientists can flip the switch to turn specific genes on or off and affect repairs on those genes.

Simple right?

No, of course not!

You need a delivery system that will safely deliver the molecular tools to the nuclei of a patient’s cells.

That’s where Wen and his team come in. Some of their earlier research looked at using high-pressured injection to deliver the CRISPR/Cas9 tool to mice with tyrosinemia.

That research showed promise in correcting the FAH gene mutation responsible for causing the body’s inability to metabolize tyrosine.

At that time the team wasn’t able to transfer the technique to clinical trials because the high pressure delivery can cause liver damage among other concerns. But by loading the genetic repair tools into some newer delivery systems going through clinical trials—including one that relies on a viral vector—the team were able to get some remarkable results. After administering the treatment to mice with Tyrosinemia, researchers found a slowdown in liver damage, and the liver was creating new, corrected FAH genes.

While human trials could be years in the future, Wen is hopeful that they create not only a treatment for Tyrosinemia type I, but that it can be tailored for other liver diseases too. And you don’t need a Nobel Prize to appreciate what that can mean to millions of people around the world.

Check out Nanotechnology Now to read the full press release.

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