Forum for Science, Industry and Business

Sponsored by:     3M 
Search our Site:

 

Gene therapy reverses muscular dystrophy in animal model

17.09.2002


Researchers have proven that gene therapy can reverse the pathological features of muscular dystrophy in an animal model. Before, gene therapy had only been able to prevent further muscle-wasting in mice. "We expect to build on these results in the continuing search for a way to treat a horrible disease. Our results indicate that gene therapy could be used not only to halt or prevent this disease, but also to restore normal muscle function in older patients," says Dr. Jeffrey S. Chamberlain, professor of neurology at the University of Washington School of Medicine in Seattle.



Chamberlain is the senior author of the paper describing the results, which will be published in the Proceedings of the National Academy of Sciences online Early Edition the week of Sept. 16 to 20.

Duchenne muscular dystrophy is an X-linked genetic disorder that strikes one of every 3,500 newborn boys. The genetic disorder means the body does not produce the dystrophin protein, which is necessary for the structural support of muscle. Without this protein, muscles weaken to the point where the victim cannot survive. There is no specific treatment against any form of muscular dystrophy, except for supportive measures, such as physical therapy, assistive technology and corrective surgery. Boys and men with the disorder usually die from respiratory failure before they can turn 25.


Researchers have been looking for many years for ways to introduce the dystrophin gene into the body of patients to replace the missing gene. In doing so, researchers have developed a strain of mice who lack the dystrophin gene. In the past, researchers have been able to insert the gene into newborn mice via adenoviral vectors. But those vectors have many viral properties, and results have been limited because adult mice and mice that have begun to develop symptoms of the disease developed a sharp host immune response that eliminated the therapeutic gene.

The results have also been limited because the dystrophin gene is large, and until now, there has not been a good way to deliver the entire gene and have it remain in the muscles of the mice. Last spring, Chamberlain and colleagues reported they were able to deliver a "micro" version of the dystrophin gene. Even the micro version appeared able to reverse the muscle-wasting process, but it was not as effective as the full gene.

However, in the Sept. 16 paper, Chamberlain and colleagues describe how they developed A stripped-down vectors that did not raise a host immune response and which delivered the full-length, muscle-specific dystrophin gene. They showed that the full gene could be delivered to muscles of young and old mice, even well after severe muscle damage had developed. Also, they showed that normal muscle function was restored to a level that directly correlated with the amount of the gene that was delivered.

"These results are extremely encouraging. We have shown that replacing the dystrophin gene will correct this disease, even in older animals. In future research, we hope to develop better methods to deliver the gene to all the muscles of the body, as currently we are limited to treating relatively small muscles. We believe these results also support the need to move forward with human clinical trials to assess the safety of these methods in patients," Chamberlain said.

The research was funded by grants from the National Institutes of Health, the Muscular Dystrophy Association and the Apex Foundation, a family foundation established by Bruce and Jolene McCaw. Other authors of the paper include Christiana DelloRusso, the lead author, of the UW Departments of Physiology and Neurology; Jeannine M. Scott, Dennis Hartigan-O’Connor and Robert W. Crawford of the Department of Neurology; former UW researchers Giovanni Salvatori, Catherine Barjot and Ann S. Robinson, and Susan V. Brooks of the University of Michigan.

Walter Neary | EurekAlert!
Further information:
http://www.washington.edu/

More articles from Health and Medicine:

nachricht Correct connections are crucial
26.06.2017 | Charité - Universitätsmedizin Berlin

nachricht One gene closer to regenerative therapy for muscular disorders
01.06.2017 | Cincinnati Children's Hospital Medical Center

All articles from Health and Medicine >>>

The most recent press releases about innovation >>>

Die letzten 5 Focus-News des innovations-reports im Überblick:

Im Focus: Can we see monkeys from space? Emerging technologies to map biodiversity

An international team of scientists has proposed a new multi-disciplinary approach in which an array of new technologies will allow us to map biodiversity and the risks that wildlife is facing at the scale of whole landscapes. The findings are published in Nature Ecology and Evolution. This international research is led by the Kunming Institute of Zoology from China, University of East Anglia, University of Leicester and the Leibniz Institute for Zoo and Wildlife Research.

Using a combination of satellite and ground data, the team proposes that it is now possible to map biodiversity with an accuracy that has not been previously...

Im Focus: Climate satellite: Tracking methane with robust laser technology

Heatwaves in the Arctic, longer periods of vegetation in Europe, severe floods in West Africa – starting in 2021, scientists want to explore the emissions of the greenhouse gas methane with the German-French satellite MERLIN. This is made possible by a new robust laser system of the Fraunhofer Institute for Laser Technology ILT in Aachen, which achieves unprecedented measurement accuracy.

Methane is primarily the result of the decomposition of organic matter. The gas has a 25 times greater warming potential than carbon dioxide, but is not as...

Im Focus: How protons move through a fuel cell

Hydrogen is regarded as the energy source of the future: It is produced with solar power and can be used to generate heat and electricity in fuel cells. Empa researchers have now succeeded in decoding the movement of hydrogen ions in crystals – a key step towards more efficient energy conversion in the hydrogen industry of tomorrow.

As charge carriers, electrons and ions play the leading role in electrochemical energy storage devices and converters such as batteries and fuel cells. Proton...

Im Focus: A unique data centre for cosmological simulations

Scientists from the Excellence Cluster Universe at the Ludwig-Maximilians-Universität Munich have establised "Cosmowebportal", a unique data centre for cosmological simulations located at the Leibniz Supercomputing Centre (LRZ) of the Bavarian Academy of Sciences. The complete results of a series of large hydrodynamical cosmological simulations are available, with data volumes typically exceeding several hundred terabytes. Scientists worldwide can interactively explore these complex simulations via a web interface and directly access the results.

With current telescopes, scientists can observe our Universe’s galaxies and galaxy clusters and their distribution along an invisible cosmic web. From the...

Im Focus: Scientists develop molecular thermometer for contactless measurement using infrared light

Temperature measurements possible even on the smallest scale / Molecular ruby for use in material sciences, biology, and medicine

Chemists at Johannes Gutenberg University Mainz (JGU) in cooperation with researchers of the German Federal Institute for Materials Research and Testing (BAM)...

All Focus news of the innovation-report >>>

Anzeige

Anzeige

Event News

Plants are networkers

19.06.2017 | Event News

Digital Survival Training for Executives

13.06.2017 | Event News

Global Learning Council Summit 2017

13.06.2017 | Event News

 
Latest News

Extensive Funding for Research on Chromatin, Adrenal Gland, and Cancer Therapy

28.06.2017 | Awards Funding

Predicting eruptions using satellites and math

28.06.2017 | Earth Sciences

Extremely fine measurements of motion in orbiting supermassive black holes

28.06.2017 | Physics and Astronomy

VideoLinks
B2B-VideoLinks
More VideoLinks >>>