Forum for Science, Industry and Business

Sponsored by:     3M 
Search our Site:

 

First ever multi-cellular model of rare disease developed at University of Alberta

23.09.2011
Research groups worldwide have tried to develop a simple model of a rare, fatal disease called Zellweger's syndrome but none has succeeded, until researchers at the Faculty of Medicine & Dentistry at the University of Alberta did so in fruit flies.

Zellweger's syndrome is a form of peroxisome biogenesis disorder, a group of deadly genetic diseases that claim the lives of children usually before they reach their first birthday. Researchers have been stumped about how to make a multicellular model they can use to development treatments.

The chair of the Department of Cell Biology, Richard Rachubinksi, and his Ph.D student Fred Mast, with the help of Drosophila [fruit fly] expert Andrew Simmonds, have been successful in developing a model of Zellweger's syndrome. This syndrome is the most common type of peroxisome biogenesis disorder.

"Mating two parents that have the mutated gene gave us a mutant fly that mimicked the human phenotype," said Rachubinski. The fruit fly is ideal for medical research because its development can be studied from fertilization through to adulthood, and the development is much more rapid than in mice or humans.

"The periods that you can allow for development are much shorter in flies so you can look at things much more quickly," said Rachubinski. "You get two generations per month."

It is also less expensive to use Drosophila. As the research group moves forward testing compounds that could be used as pharmaceuticals to treat Zellweger's syndrome, they only have to use minute amounts compared to what would be needed for other laboratory models. And it helps that part of the study included a comprehensive gene analysis that will help them monitor the efficacy of compounds and point to new gene targets for pharmaceuticals.

This is a major step forward and it has clinicians at Johns Hopkins and McGill universities excited. They have paired up with the U of A basic scientists and hope to take what the researchers learn in flies right to patients.

"We have a plan all the way to the patient," said Rachubinski. "This really is what one calls translational research. It's going from basic molecular studies, to the hopeful development of compounds, to the application in patients."

"We hope it will be a cycle, in that we feed to the clinicians information which they will then use to generate more questions," said Simmonds. "We want them to then feed the questions right back for us to work it out."

For Rachubinski, this major advance, which is published in Disease Models & Mechanisms, is a great accomplishment. He has been working to understand peroxisome biogenesis disorders for almost 30 years.

"It's what I've worked for all my life and I hope to see in the next few years that we can actually move it on even farther and look towards the treatment of these patients," he said.

Quinn Phillips | EurekAlert!
Further information:
http://www.ualberta.ca

More articles from Health and Medicine:

nachricht How cancer metastasis happens: Researchers reveal a key mechanism
19.01.2018 | Weill Cornell Medicine

nachricht Researchers identify new way to unmask melanoma cells to the immune system
17.01.2018 | Duke University 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: Artificial agent designs quantum experiments

On the way to an intelligent laboratory, physicists from Innsbruck and Vienna present an artificial agent that autonomously designs quantum experiments. In initial experiments, the system has independently (re)discovered experimental techniques that are nowadays standard in modern quantum optical laboratories. This shows how machines could play a more creative role in research in the future.

We carry smartphones in our pockets, the streets are dotted with semi-autonomous cars, but in the research laboratory experiments are still being designed by...

Im Focus: Scientists decipher key principle behind reaction of metalloenzymes

So-called pre-distorted states accelerate photochemical reactions too

What enables electrons to be transferred swiftly, for example during photosynthesis? An interdisciplinary team of researchers has worked out the details of how...

Im Focus: The first precise measurement of a single molecule's effective charge

For the first time, scientists have precisely measured the effective electrical charge of a single molecule in solution. This fundamental insight of an SNSF Professor could also pave the way for future medical diagnostics.

Electrical charge is one of the key properties that allows molecules to interact. Life itself depends on this phenomenon: many biological processes involve...

Im Focus: Paradigm shift in Paris: Encouraging an holistic view of laser machining

At the JEC World Composite Show in Paris in March 2018, the Fraunhofer Institute for Laser Technology ILT will be focusing on the latest trends and innovations in laser machining of composites. Among other things, researchers at the booth shared with the Aachen Center for Integrative Lightweight Production (AZL) will demonstrate how lasers can be used for joining, structuring, cutting and drilling composite materials.

No other industry has attracted as much public attention to composite materials as the automotive industry, which along with the aerospace industry is a driver...

Im Focus: Room-temperature multiferroic thin films and their properties

Scientists at Tokyo Institute of Technology (Tokyo Tech) and Tohoku University have developed high-quality GFO epitaxial films and systematically investigated their ferroelectric and ferromagnetic properties. They also demonstrated the room-temperature magnetocapacitance effects of these GFO thin films.

Multiferroic materials show magnetically driven ferroelectricity. They are attracting increasing attention because of their fascinating properties such as...

All Focus news of the innovation-report >>>

Anzeige

Anzeige

Event News

10th International Symposium: “Advanced Battery Power – Kraftwerk Batterie” Münster, 10-11 April 2018

08.01.2018 | Event News

See, understand and experience the work of the future

11.12.2017 | Event News

Innovative strategies to tackle parasitic worms

08.12.2017 | Event News

 
Latest News

Meteoritic stardust unlocks timing of supernova dust formation

19.01.2018 | Physics and Astronomy

Method uses DNA, nanoparticles and lithography to make optically active structures

19.01.2018 | Materials Sciences

More genes are active in high-performance maize

19.01.2018 | Life Sciences

VideoLinks
B2B-VideoLinks
More VideoLinks >>>