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.

Related Articles


"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 January 25, 2015 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 January 25, 2015).

Share This


More From ScienceDaily



More Health & Medicine News

Sunday, January 25, 2015

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

Disneyland Measles Outbreak Spreads To 5 States

Disneyland Measles Outbreak Spreads To 5 States

Newsy (Jan. 24, 2015) Much of the Disneyland measles outbreak is being blamed on the anti-vaccination movement. The CDC encourages just about everyone get immunized. Video provided by Newsy
Powered by NewsLook.com
Growing Measles Outbreak Worries Calif. Parents

Growing Measles Outbreak Worries Calif. Parents

AP (Jan. 23, 2015) Public health officials are rushing to contain a measles outbreak that has sickened 70 people across 6 states and Mexico. The AP&apos;s Raquel Maria Dillon has more. (Jan. 23) Video provided by AP
Powered by NewsLook.com
Smart Wristband to Shock Away Bad Habits

Smart Wristband to Shock Away Bad Habits

Reuters - Innovations Video Online (Jan. 23, 2015) A Boston start-up is developing a wristband they say will help users break bad habits by jolting them with an electric shock. Ben Gruber reports. Video provided by Reuters
Powered by NewsLook.com
This Is What It's Like To Date A Med Student

This Is What It's Like To Date A Med Student

BuzzFeed (Jan. 23, 2015) Dating is now speed-dating... or studying. Video provided by BuzzFeed
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:

Strange & Offbeat Stories


Health & Medicine

Mind & Brain

Living & Well

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