Researcher: Muscular Dystrophy treatment for humans 2 years away

2 years 8 months 4 weeks ago Thursday, October 22 2015 Oct 22, 2015 Thursday, October 22, 2015 12:24:00 PM CDT October 22, 2015 in News
By: Haley Hughes, KOMU 8 Reporter
loading

COLUMBIA - A researcher at the University of Missouri said Thursday he can safely say treatment for humans with Duchenne Muscular Dystrophy (DMD) is two years away. 

Dongsheng Duan said, "If you were a kid diagnosed with the disease, from the moment you get the diagnosis, you're done, that's it, no happy life. They die by the time most of us are graduating from college."

He said the initial gene mutation responsible for the disease was discovered back in 1986. It disrupts the production of a protein that protects muscles from degeneration. The new treatment works by introducing a fully functional version of the gene into the body using a harmless virus.

"At the time, they thought it would be fairly easy to put the gene back into the body, but the road to that goal is not that straightforward," Duan said.  

He said his team was able to cure a mouse improving the overall function of its muscles. 

"A mouse is a mouse. it's not a human, so to get from a mouse to a human there are several things," Duan said. "We needed to find a model between the two."  

Researchers moved on to testing the treatment in dogs.

A few years ago, he said they did an injection into a single muscle of a dog.

"A dog naturally develops DMD identical to humans and if we can treat dogs we can move this to a boy," Duan said. 

He said after the initial injection they measured the muscle force and saw considerable improvement. They then injected the virus containing the gene into the blood stream of the dog.

"We saw that there was no toxicity. We looked at [the muscle] activity, it was great. We did a biopsy and took some piece of muscle out of dog and examined it under a microscope and it looked fabulous, it's incredible," Duan said. 

He said the virus used is safe and is the only virus that can go to all of the muscles in the body.

"80 percent of us have been infected at some point by this virus, most of the time it's nothing. We don't even know we have been affected," Duan said. 

 The virus is the forefront for a number of gene therapy applications, he said.

"In one clinical trial they tried to treat a boy with a disease that kept him from seeing. They put the normal gene into the virus and the boy could see," Duan said. 

He said treatment for DMD will soon be as simple as getting an injection.

"It would keep the remaining muscle healthy and improve their life quality. They don't go downhill, but rather maintain stable [muscle function] or even go upward," Duan said. 

He said is he hopeful.

"We are finally getting to a stage where we can say we are getting pretty close, it's going to happen," he said.

 

 

 

More News

Grid
List
WASHINGTON (AP) — President Donald Trump's choice for the Supreme Court has given members of Congress a pile of material... More >>
3 hours ago Saturday, July 21 2018 Jul 21, 2018 Saturday, July 21, 2018 2:52:51 PM CDT July 21, 2018 in News
WASHINGTON (AP) — The maker of a permanent contraceptive implant subject to thousands of injury reports and repeated safety restrictions... More >>
4 hours ago Saturday, July 21 2018 Jul 21, 2018 Saturday, July 21, 2018 2:28:05 PM CDT July 21, 2018 in News
(CNN) -- The man accused of carrying out last month's deadly shooting at a Maryland newspaper has been indicted on... More >>
4 hours ago Saturday, July 21 2018 Jul 21, 2018 Saturday, July 21, 2018 1:42:30 PM CDT July 21, 2018 in News
COLUMBIA - The 2018 Show-Me State Games kick off Friday, bringing thousands of athletes, coaches and fans from around the... More >>
5 hours ago Saturday, July 21 2018 Jul 21, 2018 Saturday, July 21, 2018 1:15:00 PM CDT July 21, 2018 in News
ASHLAND – Seven locations throughout the state are open Saturday for parents to come and get their children I.D.’d, free... More >>
5 hours ago Saturday, July 21 2018 Jul 21, 2018 Saturday, July 21, 2018 12:58:00 PM CDT July 21, 2018 in News
(CNN) -- Gracelyn Mae Violet Griffin already had a "Little Nugget" onesie picked out for her before she arrived in... More >>
5 hours ago Saturday, July 21 2018 Jul 21, 2018 Saturday, July 21, 2018 12:35:28 PM CDT July 21, 2018 in News
JEFFERSON CITY (AP) — An organization backing one of three competing medical marijuana initiatives in Missouri has continued to bring... More >>
7 hours ago Saturday, July 21 2018 Jul 21, 2018 Saturday, July 21, 2018 11:26:00 AM CDT July 21, 2018 in News
LAKE OF THE OZARKS - 64 years later, a different lake and nearly triple the death toll, the duck boat... More >>
21 hours ago Friday, July 20 2018 Jul 20, 2018 Friday, July 20, 2018 9:12:00 PM CDT July 20, 2018 in News
(CNN) -- Russian intelligence operatives attempted to hack into the online accounts of staffers on three congressional campaigns in the... More >>
21 hours ago Friday, July 20 2018 Jul 20, 2018 Friday, July 20, 2018 8:46:27 PM CDT July 20, 2018 in News
CHARLOTTE, N.C. (AP) — As Charlotte, North Carolina, celebrates being chosen Friday to host the 2020 Republican National Convention, an... More >>
22 hours ago Friday, July 20 2018 Jul 20, 2018 Friday, July 20, 2018 8:05:32 PM CDT July 20, 2018 in News
(CNN) -- A cardiologist who treated former President George H.W. Bush was killed Friday morning in a bicycle-to-bicycle drive-by shooting... More >>
22 hours ago Friday, July 20 2018 Jul 20, 2018 Friday, July 20, 2018 7:34:02 PM CDT July 20, 2018 in News
COLUMBIA - The Columbia Police Department will move into a new neighborhood over the next few weeks. In July,... More >>
23 hours ago Friday, July 20 2018 Jul 20, 2018 Friday, July 20, 2018 6:41:00 PM CDT July 20, 2018 in News
CALLAWAY COUNTY - A Steedman man was sentenced to 12 years in prison after pleading guilty to charges of domestic... More >>
1 day ago Friday, July 20 2018 Jul 20, 2018 Friday, July 20, 2018 5:22:00 PM CDT July 20, 2018 in News
ST. JOSEPH (AP) — A Missouri man has pleaded guilty in the fatal beating of his 77-year-old grandmother while he... More >>
1 day ago Friday, July 20 2018 Jul 20, 2018 Friday, July 20, 2018 3:59:00 PM CDT July 20, 2018 in News
(CNN) -- The FBI is in possession of a recording between President Donald Trump and his former personal attorney Michael... More >>
1 day ago Friday, July 20 2018 Jul 20, 2018 Friday, July 20, 2018 3:38:16 PM CDT July 20, 2018 in Continuous News
JEFFERSON CITY – House Minority Leader Gail McCann Beatty will resign from the Missouri House of Representatives effective Sunday in... More >>
1 day ago Friday, July 20 2018 Jul 20, 2018 Friday, July 20, 2018 12:57:00 PM CDT July 20, 2018 in Top Stories
COLE COUNTY — The Cole County Sheriff’s Department responded to possible weapons violation on Thursday night. When the officers... More >>
1 day ago Friday, July 20 2018 Jul 20, 2018 Friday, July 20, 2018 11:47:00 AM CDT July 20, 2018 in Top Stories
(AP) - President Donald Trump has indicated that he's willing to hit every product imported from China with tariffs and... More >>
1 day ago Friday, July 20 2018 Jul 20, 2018 Friday, July 20, 2018 8:52:00 AM CDT July 20, 2018 in News
Columbia, MO
Broken Clouds 87°
7pm 85°
8pm 83°
9pm 80°
10pm 77°