Click to Translate to English Click to Translate to French  Click to Translate to Spanish  Click to Translate to German  Click to Translate to Italian  Click to Translate to Japanese  Click to Translate to Chinese Simplified  Click to Translate to Korean  Click to Translate to Arabic  Click to Translate to Russian  Click to Translate to Portuguese  Click to Translate to Myanmar (Burmese)

PANDEMIC ALERT LEVEL
123456
Forum Home Forum Home > Main Forums > General Discussion
  New Posts New Posts RSS Feed - Early Days, but REAL Hope for Huntingtons
  FAQ FAQ  Forum Search   Events   Register Register  Login Login

Now tracking the new emerging South Africa Omicron Variant

Early Days, but REAL Hope for Huntingtons

 Post Reply Post Reply
Author
Message
Technophobe View Drop Down
Assistant Admin
Assistant Admin
Avatar

Joined: January 16 2014
Location: Scotland
Status: Offline
Points: 88450
Post Options Post Options   Thanks (0) Thanks(0)   Quote Technophobe Quote  Post ReplyReply Direct Link To This Post Topic: Early Days, but REAL Hope for Huntingtons
    Posted: June 20 2017 at 4:14pm

Scientists Used CRISPR to Reverse Huntington's Disease in Mice

The gene-editing technique CRISPR is often touted as an eventual cure-all for all that ails us, from fatal genetic diseases to food shortages. But when it comes to disease, it’s likely that it will have the most impact on disorders caused by mutations in one single gene. New research published this week in the Journal of Clinical Investigation suggests that Huntington’s Disease may be a good candidate for a CRISPR cure.

Huntington’s Disease is a fatal, inherited disorder that gradually causes the breakdown of nerve cells in the brain. It’s caused by a gene that encodes a toxic protein that causes brain cells to die, with symptoms that usually show up around mid life.

In the new study from scientists at Emory University, researchers used mice engineered to possess the same human gene that causes Huntington’s. In such mice, the motor problems associated with Huntington’s show up at about 9 months of age. They then used CRISPR-Cas9 to snip the part of the gene that produces the toxic protein. Weeks later, the researchers found that those proteins had almost fully disappeared from the brain, and the motor abilities of the mice had improved (though they still had some motor impairment). Longer term effects of the treatment are not yet clear, although the researchers think the genetic alteration will be permanent.

Their findings, the researchers wrote, suggested a permanent therapeutic treatment for Huntington’s, and potentially other neurodegenerative diseases, too.

This research, of course, is preliminary, though other scientists have said it isencouraging.

Importantly, in their paper the scientists reported finding no off-target effects, a major concern associated with using CRISPR in humans. Still, they write, it will have to be tested for long-term safety and efficacy before being ready to try out in people.

Already, there are two clinical trials using CRISPR in humans underfoot in China, and a US trial is slated to begin sometime in the next year. But those trials are aimed at cancer. While scientists have had success treating other single-mutation conditions like sickle-cell anaemia and blindness in mice, so far, human trials for such diseases have not begun.

[Journal of Clinical Investigation]

Source:  http://gizmodo.com/scientists-used-crispr-to-reverse-huntingtons-disease-i-1796262804

How do you tell if a politician is lying?
His lips or pen are moving.
Back to Top
 Post Reply Post Reply
  Share Topic   

Forum Jump Forum Permissions View Drop Down