This Huntington's disease trial shows how promising gene editing may be.

Huntington’s disease is caused by a single mutation in someone’s DNA.

A normal protein, huntingtin, becomes warped and toxic. Over time, the toxic protein builds up, killing a person’s brain cells and slowly robbing them of cognitive and motor control.

It affects about 30,000 Americans, often shows up later in life, and is fatal. Because it’s a genetic disease, it can be passed down through families. It’s cruel, unfair, and nobody deserves it.

But what if we could just zap away that one fatal mutation?

That’s exactly what a team of scientists from Emory University and the Chinese Academy of Sciences just tried — zapping that mutation right out of the DNA. And it looks like it could work.

Working with sick mice in a lab, the scientists used a gene-editing technique colloquially known as CRISPR. On June 19, 2017, the scientists reported that once injected and inside the brain cells, the CRISPR system could read the mice’s DNA, find the huntingtin gene, and snip it apart. This effectively silenced the gene, cutting off the “flow” of the toxic proteins.

When the scientists checked on the mice a few weeks later, the toxic proteins had nearly disappeared from their cells. The scientists reported that the sick mice also regained some, but not all, of their motor control.

This isn’t the first trial to use gene editing on Huntington’s, but it does represent a new technique.

There’s currently no cure for this awful disease, but this kind of research could provide a path to one in the future.

Gene editing is still pretty new; we need to learn more about whether CRISPR is completely safe, for instance, or what the long-term effects of silencing that particular gene would be.

But if we look at the history of humanity, we’ve conquered many horrible diseases. We’ve beaten back polio and smallpox and are even tackling childhood cancer. It’s not far-fetched to think that, one day, we may be able to add Huntington’s to the list.





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