HOME >> BIOLOGY >> NEWS
Duke scientists reverse a rare form of muscular dystrophy in mice using gene therapy

DURHAM, N.C. -- Using a modified virus to deliver a therapeutic gene, scientists at Duke University Medical Center have shown that, in mice, they can reverse the damage caused by an inherited muscle-wasting disease with a single injected dose.

The study findings, which appear in the Aug. 3 issue of the Proceedings of the National Academy of Science, show for the first time that it appears possible to deliver a therapeutic gene product throughout all of the muscles of the body to reverse muscle wasting, a result that has implications for treating dozens of forms of muscular dystrophy.

The researchers note, however, that to date they have only demonstrated a short-term reversal of symptoms in laboratory mice, and further experiments are needed to determine if the approach could become practical for use in people.

The study is part of a large, collaborative effort at Duke University to find an effective treatment for Pompe disease, a rare inherited disorder in which the body can not process glycogen, a stored form of sugar the body needs for energy. People born with Pompe disease have a defect in an enzyme called acid alpha-glucosidase (GAA), which normally processes glycogen and converts it to sugar. The glycogen builds up in muscle tissues throughout the body, including the heart, causing the various muscles to degenerate.

Several forms of Pompe disease affect more than 5,000 people in the United States. If symptoms appear during infancy, the disease is usually fatal. It is usually less severe when symptoms first appear late in childhood, but life expectancy is greatly deceased. Although Pompe disease is a relatively rare disease, it is but one of a group of lysosomal storage diseases, which in total occur in about one in 5,000 births in the United States.

Duke pediatric geneticist Y. T. Chen has been simultaneously pursuing two avenues to treating the disease: replacing the missing GAA en
'"/>

Contact: Karyn Hede
Hede0001@mc.duke.edu
919-684-4148
Duke University Medical Center
2-Aug-1999


Page: 1 2 3

Related biology news :

1. DNA lends scientists a hand, revealing new chemical reactions
2. Conference at UH opens doors for new scientists, engineers
3. Wisconsin scientists develop quick botox test
4. UCI scientists successfully target key HIV protein; breakthrough may lead to new drug therapies
5. Alaska scientists find Arctic tundra yields surprising carbon loss
6. UAF scientists discover new marine habitat in Alaska
7. Information system to help scientists analyze mechanisms of social behavior
8. Zoonotic diseases - European scientists unite to fight diseases
9. Israeli scientists reveal the plan of a key cellular machine
10. Study by Israeli scientists provides insight on DNA code
11. Computer scientists at UH developing nurturing computers

Post Your Comments:
*Name:
*Comment:
*Email:


(Date:8/12/2020)... ... August 11, 2020 , ... ... in Princeton, NJ, have entered into license agreements with Housey Pharma’s HMI subsidiary ... Both Roche and J&J have annual Research and Development spending in excess of ...
(Date:8/5/2020)... , ... August 05, 2020 , ... Regenative Labs has ... new innovations, CoreText™ and ProText™, making them the first Wharton’s jelly allografts to be ... a syringe. The company’s solutions are the first Wharton’s jelly allograft product to be ...
(Date:7/22/2020)... Calif. (PRWEB) , ... July 21, 2020 , ... ... driving digital transformation and innovation in technology and compliance, announces a new solution ... device companies must ensure every layer of their technology stack complies with FDA ...
Breaking Biology News(10 mins):
(Date:7/31/2020)... ... July 29, 2020 , ... eSource has long been touted as ... cover the history of eSource, the reasons it did not take off as quickly ... site source, the industry is moving towards capturing data electronically for clinical trials and ...
(Date:7/22/2020)... ... July 22, 2020 , ... Join experts from Reed Tech , Gary ... in a one hour live webinar on Thursday, August 13, 2020 at ... drugs and medical devices. Specifically, for medical devices, the NMPA has departments dealing with ...
(Date:7/18/2020)... ... July 16, 2020 , ... Medial ... early detection and prevention of high-burden diseases, and Centric Consulting, a business and ... organizations to utilize existing data in order to identify and prioritize patients for ...
(Date:7/10/2020)... (PRWEB) , ... July 09, ... ... biotechnology company, announced today that Massachusetts Institute of Technology (MIT) has expanded ... broader license allows PathSensors to move into the point-of-care diagnostic market, focusing ...
Breaking Biology Technology:
Cached News: