Researcher: Muscular Dystrophy treatment for humans 2 years away

3 years 10 months 3 days 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
JEFFERSON CITY- The Long-Term Recovery committee is looking for volunteers to help with disaster relief in Cole and Miller Counties.... More >>
1 hour ago Monday, August 26 2019 Aug 26, 2019 Monday, August 26, 2019 2:54:00 AM CDT August 26, 2019 in News
COLUMBIA - Boonville School District buses will take snow routes Monday due to flooding. According to Boonville School District,... More >>
5 hours ago Sunday, August 25 2019 Aug 25, 2019 Sunday, August 25, 2019 11:14:00 PM CDT August 25, 2019 in News
COLUMBIA - As Rock Bridge High School student Hope Keithahn headed into her senior year, she came up with an... More >>
5 hours ago Sunday, August 25 2019 Aug 25, 2019 Sunday, August 25, 2019 10:30:00 PM CDT August 25, 2019 in News
COLUMBIA — Troopers said a Fulton teenager was seriously injured after she was ejected from her car in a Callaway... More >>
7 hours ago Sunday, August 25 2019 Aug 25, 2019 Sunday, August 25, 2019 8:18:00 PM CDT August 25, 2019 in News
OVERTON, Mo. - Authorities are searching for a motorcycle rider who fled the scene of a crash on I-70 Sunday.... More >>
9 hours ago Sunday, August 25 2019 Aug 25, 2019 Sunday, August 25, 2019 6:51:00 PM CDT August 25, 2019 in News
COLUMBIA – One of the nation’s most restrictive abortion laws is scheduled to go into effect in Missouri on Aug.... More >>
10 hours ago Sunday, August 25 2019 Aug 25, 2019 Sunday, August 25, 2019 5:32:00 PM CDT August 25, 2019 in News
COLUMBIA - A new exhibit, organized by the League of Woman Voters and the Boone County History and Culture Center,... More >>
11 hours ago Sunday, August 25 2019 Aug 25, 2019 Sunday, August 25, 2019 5:05:00 PM CDT August 25, 2019 in News
HIGBEE - After closing last June, the Higbee Senior Center is attempting to re-open. The center hosted a benefit lunch... More >>
11 hours ago Sunday, August 25 2019 Aug 25, 2019 Sunday, August 25, 2019 4:19:00 PM CDT August 25, 2019 in Continuous News
SUNRISE BEACH - The Lake of the Ozarks Shootout resumed Sunday after being suspended Saturday. The race was called... More >>
12 hours ago Sunday, August 25 2019 Aug 25, 2019 Sunday, August 25, 2019 3:31:00 PM CDT August 25, 2019 in News
COLUMBIA - After five years of a growing business, Peggy Jean's Pies is moving to a new location. With... More >>
1 day ago Saturday, August 24 2019 Aug 24, 2019 Saturday, August 24, 2019 8:05:00 PM CDT August 24, 2019 in News
ASHLAND - After Gov. Mike Parson called for the state legislature to convene in a special session to discuss a... More >>
1 day ago Saturday, August 24 2019 Aug 24, 2019 Saturday, August 24, 2019 6:46:00 PM CDT August 24, 2019 in News
INDIANAPOLIS (AP) — A defense attorney is seeking a psychiatric exam for the semi driver charged with causing a highway... More >>
1 day ago Saturday, August 24 2019 Aug 24, 2019 Saturday, August 24, 2019 4:06:00 PM CDT August 24, 2019 in News
LAKE OF THE OZARKS — Missouri State Highway Patrol divers recovered the body of a missing man Saturday night from... More >>
1 day ago Saturday, August 24 2019 Aug 24, 2019 Saturday, August 24, 2019 3:50:00 PM CDT August 24, 2019 in News
DES MOINES, Iowa (AP) — An Iowa-based grocery chain says it's aware of reports that hacked customer account information is... More >>
1 day ago Saturday, August 24 2019 Aug 24, 2019 Saturday, August 24, 2019 2:23:00 PM CDT August 24, 2019 in News
COLUMBIA - Columbia police are investigating a crash on I-70 at Stadium Boulevard on Saturday afternoon that left one person... More >>
1 day ago Saturday, August 24 2019 Aug 24, 2019 Saturday, August 24, 2019 1:50:00 PM CDT August 24, 2019 in News
COLUMBIA - Participants raised $83,988 for the Columbia Walk to Defeat ALS on Saturday. In its 12th year, the... More >>
1 day ago Saturday, August 24 2019 Aug 24, 2019 Saturday, August 24, 2019 1:48:00 PM CDT August 24, 2019 in News
COLUMBIA - A butterfly mural has appeared in a Columbia alley and has become a new photo destination. Children's... More >>
1 day ago Saturday, August 24 2019 Aug 24, 2019 Saturday, August 24, 2019 11:35:00 AM CDT August 24, 2019 in News
COLUMBIA, Mo. (AP) — Four years after student protesters denouncing a racist campus culture and demanding a more diverse faculty... More >>
1 day ago Saturday, August 24 2019 Aug 24, 2019 Saturday, August 24, 2019 10:26:00 AM CDT August 24, 2019 in News
Columbia, MO
Broken Clouds 70°
5am 68°
6am 67°
7am 70°
8am 71°