Latest in Tomorrow

Image credit: from2015 via Getty Images

Gene editing technique could treat ALS and Huntington's disease

It might delay the onset of conditions that are normally inescapable.
1200 Shares
Share
Tweet
Share
Save

Sponsored Links

from2015 via Getty Images

The most common gene editing technique, CRISPR-Cas9, only modifies DNA. That's helpful in most cases, but it means that you can't use it to tackle RNA-based diseases. Thankfully, that might not be a problem for much longer. After plenty of talk about editing RNA, researchers have developed a new RNA-oriented technique (RCas9) that can correct the molecular errors which lead to diseases like hereditary ALS and Huntington's.

The team achieved its feat using guide RNA that steers the editing enzyme toward a matching sequence of target RNA molecules. In the lab, it's highly effective -- it fixed nearly all of the broken RNA targets in muscle cells, leaving cells that were far healthier.

Of course, this is in lab conditions. The scientists readily admit that there's much more work to be done before gene editing could help in the field. More than anything, it would be difficult to send RCas9 to patient cells. The benign viruses you'd normally use for gene therapy just can't hold a full-fledged Cas9 payload; the researchers had to prune theirs to make it fit. And of course, there's the question of conducting trials and proving that the technique is safe.

Nonetheless, this could prove to be hugely important for treating these RNA diseases. Right now, there aren't any therapies that would stall the onset of diseases like ALS. This wouldn't likely represent a cure, but it could give patients full functionality for longer before the effects of their diseases set in.

All products recommended by Engadget are selected by our editorial team, independent of our parent company. Some of our stories include affiliate links. If you buy something through one of these links, we may earn an affiliate commission.
Comment
Comments
Share
1200 Shares
Share
Tweet
Share
Save

Popular on Engadget

AI can gauge the risk of dying from heart conditions

AI can gauge the risk of dying from heart conditions

View
OnePlus 7T Pro may debut on October 10th

OnePlus 7T Pro may debut on October 10th

View
'Minecraft' now has 112 million players per month

'Minecraft' now has 112 million players per month

View
Central banks to question Facebook over Libra cryptocurrency

Central banks to question Facebook over Libra cryptocurrency

View
Verizon will launch home 5G everywhere mobile service is available

Verizon will launch home 5G everywhere mobile service is available

View

From around the web

Page 1Page 1ear iconeye iconFill 23text filevr