Featured Research

from universities, journals, and other organizations

Effects Of Muscular Dystrophy Reversed By Gene Therapy, Reports Team From Children's National Medical Center And The University Of Pittsburgh

Date:
October 27, 1999
Source:
University Of Pittsburgh Medical Center
Summary:
Animal muscles crippled due to a form of muscular dystrophy can be repaired, both in size and strength, through an innovative gene therapy, according to a team of investigators from the Children's National Medical Center in Washington, D.C., and the University of Pittsburgh.

SAN FRANCISCO, Oct. 19 -- Animal muscles crippled due to a form of muscular dystrophy can be repaired, both in size and strength, through an innovative gene therapy, according to a team of investigators from the Children's National Medical Center in Washington, D.C., and the University of Pittsburgh. The results, presented Friday, Oct. 22, at the annual meeting of the American Society of Human Genetics (ASHG) in San Francisco, offer the first evidence that whole muscles can be functionally rescued in any type of muscular dystrophy using an extremely safe gene vector.

"We are very excited by these preliminary results, which suggest that we can use a non-toxic virus to safely shuttle a gene for an important muscle protein that is improperly made in people suffering limb girdle muscular dystrophy," said Devin Dressman, who is presenting the results and who is a graduate student at the Center for Genetic Medicine, Children's National Medical Center, and the University of Pittsburgh's School of Medicine. "These findings demonstrate the feasibility of using the gene therapy approach in treating affected patients."

In their experiments, the investigators used a non-replicating adeno-associated virus (AAV) to carry a gene for the sarcoglycan protein, an important constituent of skeletal muscle. Unlike several other available viral vectors, AAV does not provoke an immune response from the body, either by itself or when it resides within cells. Furthermore, it can infect non-dividing cells and it takes up residence permanently, thus allowing long-term production of a critical protein.

In a collaborative effort, Xiao Xiao, Ph.D., assistant professor of molecular genetics and biochemistry at the University of Pittsburgh, injected this AAV-sarcoglycan gene combination into hamster leg muscles ravaged by limb girdle dystrophy. After one month, Jon Watchko, M.D., associate professor of pediatrics and of obstetrics, gynecology and reproductive sciences at the University of Pittsburgh, tested the treated muscles and found that they increased nearly 100 percent in strength and resumed normal size.

Limb girdle muscular dystrophy affects several thousand people in the United States, causing rapid degeneration of the large muscles attached to the shoulders and hips. Currently no effective treatment exists for this fatal disorder. The Children's/Pitt team of investigators is currently planning a gene therapy treatment protocol based on the experimental findings. This approach may be applied to other forms of muscular dystrophy as well, such as the more common lethal condition, Duchenne muscular dystrophy, which affects 20,000 boys in this country and is considered one of the most prevalent diseases to result from a single gene defect.


Story Source:

The above story is based on materials provided by University Of Pittsburgh Medical Center. Note: Materials may be edited for content and length.


Cite This Page:

University Of Pittsburgh Medical Center. "Effects Of Muscular Dystrophy Reversed By Gene Therapy, Reports Team From Children's National Medical Center And The University Of Pittsburgh." ScienceDaily. ScienceDaily, 27 October 1999. <www.sciencedaily.com/releases/1999/10/991027074645.htm>.
University Of Pittsburgh Medical Center. (1999, October 27). Effects Of Muscular Dystrophy Reversed By Gene Therapy, Reports Team From Children's National Medical Center And The University Of Pittsburgh. ScienceDaily. Retrieved August 30, 2014 from www.sciencedaily.com/releases/1999/10/991027074645.htm
University Of Pittsburgh Medical Center. "Effects Of Muscular Dystrophy Reversed By Gene Therapy, Reports Team From Children's National Medical Center And The University Of Pittsburgh." ScienceDaily. www.sciencedaily.com/releases/1999/10/991027074645.htm (accessed August 30, 2014).

Share This




More Health & Medicine News

Saturday, August 30, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

We've Got Mites Living In Our Faces And So Do You

We've Got Mites Living In Our Faces And So Do You

Newsy (Aug. 30, 2014) A new study suggests 100 percent of adult humans (those over 18 years of age) have Demodex mites living in their faces. Video provided by Newsy
Powered by NewsLook.com
Liberia Continues Fight Against Ebola

Liberia Continues Fight Against Ebola

AFP (Aug. 30, 2014) Authorities in Liberia try to stem the spread of the Ebola epidemic by raising awareness and setting up sanitation units for people to wash their hands. Duration: 00:41 Video provided by AFP
Powered by NewsLook.com
California Passes 'yes-Means-Yes' Campus Sexual Assault Bill

California Passes 'yes-Means-Yes' Campus Sexual Assault Bill

Reuters - US Online Video (Aug. 30, 2014) California lawmakers pass a bill requiring universities to adopt "affirmative consent" language in their definitions of consensual sex, part of a nationwide drive to curb sexual assault on campuses. Linda So reports. Video provided by Reuters
Powered by NewsLook.com
3 Things To Know About The Ebola Outbreak's Progression

3 Things To Know About The Ebola Outbreak's Progression

Newsy (Aug. 29, 2014) Here are three things you need to know about the deadly Ebola outbreak's progression this week. Video provided by Newsy
Powered by NewsLook.com

Search ScienceDaily

Number of stories in archives: 140,361

Find with keyword(s):
Enter a keyword or phrase to search ScienceDaily for related topics and research stories.

Save/Print:
Share:

Breaking News:
from the past week

In Other News

... from NewsDaily.com

Science News

Health News

Environment News

Technology News



Save/Print:
Share:

Free Subscriptions


Get the latest science news with ScienceDaily's free email newsletters, updated daily and weekly. Or view hourly updated newsfeeds in your RSS reader:

Get Social & Mobile


Keep up to date with the latest news from ScienceDaily via social networks and mobile apps:

Have Feedback?


Tell us what you think of ScienceDaily -- we welcome both positive and negative comments. Have any problems using the site? Questions?
Mobile: iPhone Android Web
Follow: Facebook Twitter Google+
Subscribe: RSS Feeds Email Newsletters
Latest Headlines Health & Medicine Mind & Brain Space & Time Matter & Energy Computers & Math Plants & Animals Earth & Climate Fossils & Ruins