Forum for Science, Industry and Business

Sponsored by:     3M 
Search our Site:

 

Gene therapy for muscular dystrophy fixes frail muscle cells in animal model

28.12.2005


A new gene therapy technique that has shown promise in skin disease and hemophilia might one day be useful for treating muscular dystrophy, according to a new study by researchers at Stanford University School of Medicine.



In the study, scheduled to be published online in the Proceedings of the National Academy of Sciences the week of Jan. 2, the researchers used gene therapy to introduce a healthy copy of the gene dystrophin into mice with a condition that mimics muscular dystrophy. The dystrophin gene is mutated and as a result produces a defective protein in the roughly 20,000 people in the United States with the most common form of the disease.

Using gene therapy to treat muscular dystrophy isn’t a new idea. Thomas Rando, MD, PhD, associate professor of neurology and neurological sciences, said that researchers have tried several different techniques with variable success. One hurdle is getting genes into muscle cells all over the body. Another is convincing those cells to permanently produce the therapeutic protein made by those genes.


The gene therapy technique Rando and postdoctoral fellow Carmen Bertoni, PhD, used was developed by Michele Calos, PhD, associate professor of genetics. One of the main advantages of this method is that it could potentially provide a long-term fix for a variety of genetic diseases, including muscular dystrophy.

In muscular dystrophy, the muscle cells break down and are slowly replaced by fat. Eventually people with the disease are confined to a wheelchair and usually die in their 20s. There is currently no effective treatment for the disease, which explains why gene therapy remains a hope despite the significant hurdles.

Rando said the PNAS paper highlights an additional requirement for any gene therapy to be successful: the introduced gene must produce healthy dystrophin protein in large quantities in order to repair the entire muscle cell. Previous muscular dystrophy gene therapy studies did not look at whether the introduced dystrophin spread along the entire length of the muscle cell, which can be many millimeters long in mice or inches long in humans.

In the upcoming paper Bertoni used a standard gene therapy method to introduce two genes - dystrophin and a gene that makes a glowing protein - into mice with a mouse version of muscular dystrophy. She found that in mice producing insufficient dystrophin, she could see the glowing protein slowly leak out of the cell. This leakiness is a sign that the cell is not healed. In contrast, when she used Calos’ gene therapy technique to introduce the genes, the muscle cell contained high levels of dystrophin distributed along the length of the cell and the glowing protein stayed within the cell, suggesting that the abundant dystrophin repaired the ailing muscle.

"If you have a single cell that’s a foot long and you only correct a few inches, you’ve done very little," Rando said, "Whereas if you correct it from end to end, you truly cure the disease in that cell."

Both Rando and Calos point out that the road to a gene therapy cure for muscular dystrophy is still a long one. However, Calos is confident that her technique will be a part of the journey towards a cure for the disease and for other diseases such as hemophilia and the skin disease, epidermolysis bullosa. Early trials using her approach have looked promising in animal models of both of these diseases.

"I think our approach has a lot of potential to overcome issues that have slowed the field of gene therapy," Calos said.

Calos said her approach has two advantages: one is that in her method the gene gets inserted directly into the cell’s own DNA, which is why the correction is permanent. In some other methods the gene stays outside the DNA and slowly breaks down. The second advantage is that her method doesn’t rely on a virus to disperse the DNA and therefore avoids some of the issues, including cancer and an immune reaction, that have turned up in viral gene therapy trials. Instead this approach uses naked DNA that travels through the bloodstream to cells of the body.

For his part, Rando said that no matter how well gene therapy works in an isolated muscle, researchers still must figure out how to get that gene to muscles throughout the body. Despite the remaining hurdles, both Rando and Calos said that their study is a step towards eventually treating muscular dystrophy and other diseases using gene therapy.

Amy Adams | EurekAlert!
Further information:
http://www.stanford.edu

More articles from Life Sciences:

nachricht Discovery of a Key Regulatory Gene in Cardiac Valve Formation
24.05.2017 | Universität Basel

nachricht Carcinogenic soot particles from GDI engines
24.05.2017 | Empa - Eidgenössische Materialprüfungs- und Forschungsanstalt

All articles from Life Sciences >>>

The most recent press releases about innovation >>>

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

Im Focus: A quantum walk of photons

Physicists from the University of Würzburg are capable of generating identical looking single light particles at the push of a button. Two new studies now demonstrate the potential this method holds.

The quantum computer has fuelled the imagination of scientists for decades: It is based on fundamentally different phenomena than a conventional computer....

Im Focus: Turmoil in sluggish electrons’ existence

An international team of physicists has monitored the scattering behaviour of electrons in a non-conducting material in real-time. Their insights could be beneficial for radiotherapy.

We can refer to electrons in non-conducting materials as ‘sluggish’. Typically, they remain fixed in a location, deep inside an atomic composite. It is hence...

Im Focus: Wafer-thin Magnetic Materials Developed for Future Quantum Technologies

Two-dimensional magnetic structures are regarded as a promising material for new types of data storage, since the magnetic properties of individual molecular building blocks can be investigated and modified. For the first time, researchers have now produced a wafer-thin ferrimagnet, in which molecules with different magnetic centers arrange themselves on a gold surface to form a checkerboard pattern. Scientists at the Swiss Nanoscience Institute at the University of Basel and the Paul Scherrer Institute published their findings in the journal Nature Communications.

Ferrimagnets are composed of two centers which are magnetized at different strengths and point in opposing directions. Two-dimensional, quasi-flat ferrimagnets...

Im Focus: World's thinnest hologram paves path to new 3-D world

Nano-hologram paves way for integration of 3-D holography into everyday electronics

An Australian-Chinese research team has created the world's thinnest hologram, paving the way towards the integration of 3D holography into everyday...

Im Focus: Using graphene to create quantum bits

In the race to produce a quantum computer, a number of projects are seeking a way to create quantum bits -- or qubits -- that are stable, meaning they are not much affected by changes in their environment. This normally needs highly nonlinear non-dissipative elements capable of functioning at very low temperatures.

In pursuit of this goal, researchers at EPFL's Laboratory of Photonics and Quantum Measurements LPQM (STI/SB), have investigated a nonlinear graphene-based...

All Focus news of the innovation-report >>>

Anzeige

Anzeige

Event News

Marine Conservation: IASS Contributes to UN Ocean Conference in New York on 5-9 June

24.05.2017 | Event News

AWK Aachen Machine Tool Colloquium 2017: Internet of Production for Agile Enterprises

23.05.2017 | Event News

Dortmund MST Conference presents Individualized Healthcare Solutions with micro and nanotechnology

22.05.2017 | Event News

 
Latest News

Physicists discover mechanism behind granular capillary effect

24.05.2017 | Physics and Astronomy

Measured for the first time: Direction of light waves changed by quantum effect

24.05.2017 | Physics and Astronomy

Marine Conservation: IASS Contributes to UN Ocean Conference in New York on 5-9 June

24.05.2017 | Event News

VideoLinks
B2B-VideoLinks
More VideoLinks >>>