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 Study suggests possible new target for treating and preventing Alzheimer's
02.12.2016 | Oregon Health & Science University

nachricht The first analysis of Ewing's sarcoma methyloma opens doors to new treatments
01.12.2016 | IDIBELL-Bellvitge Biomedical Research Institute

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: Shape matters when light meets atom

Mapping the interaction of a single atom with a single photon may inform design of quantum devices

Have you ever wondered how you see the world? Vision is about photons of light, which are packets of energy, interacting with the atoms or molecules in what...

Im Focus: Novel silicon etching technique crafts 3-D gradient refractive index micro-optics

A multi-institutional research collaboration has created a novel approach for fabricating three-dimensional micro-optics through the shape-defined formation of porous silicon (PSi), with broad impacts in integrated optoelectronics, imaging, and photovoltaics.

Working with colleagues at Stanford and The Dow Chemical Company, researchers at the University of Illinois at Urbana-Champaign fabricated 3-D birefringent...

Im Focus: Quantum Particles Form Droplets

In experiments with magnetic atoms conducted at extremely low temperatures, scientists have demonstrated a unique phase of matter: The atoms form a new type of quantum liquid or quantum droplet state. These so called quantum droplets may preserve their form in absence of external confinement because of quantum effects. The joint team of experimental physicists from Innsbruck and theoretical physicists from Hannover report on their findings in the journal Physical Review X.

“Our Quantum droplets are in the gas phase but they still drop like a rock,” explains experimental physicist Francesca Ferlaino when talking about the...

Im Focus: MADMAX: Max Planck Institute for Physics takes up axion research

The Max Planck Institute for Physics (MPP) is opening up a new research field. A workshop from November 21 - 22, 2016 will mark the start of activities for an innovative axion experiment. Axions are still only purely hypothetical particles. Their detection could solve two fundamental problems in particle physics: What dark matter consists of and why it has not yet been possible to directly observe a CP violation for the strong interaction.

The “MADMAX” project is the MPP’s commitment to axion research. Axions are so far only a theoretical prediction and are difficult to detect: on the one hand,...

Im Focus: Molecules change shape when wet

Broadband rotational spectroscopy unravels structural reshaping of isolated molecules in the gas phase to accommodate water

In two recent publications in the Journal of Chemical Physics and in the Journal of Physical Chemistry Letters, researchers around Melanie Schnell from the Max...

All Focus news of the innovation-report >>>

Anzeige

Anzeige

Event News

ICTM Conference 2017: Production technology for turbomachine manufacturing of the future

16.11.2016 | Event News

Innovation Day Laser Technology – Laser Additive Manufacturing

01.11.2016 | Event News

#IC2S2: When Social Science meets Computer Science - GESIS will host the IC2S2 conference 2017

14.10.2016 | Event News

 
Latest News

IHP presents the fastest silicon-based transistor in the world

05.12.2016 | Power and Electrical Engineering

InLight study: insights into chemical processes using light

05.12.2016 | Materials Sciences

High-precision magnetic field sensing

05.12.2016 | Power and Electrical Engineering

VideoLinks
B2B-VideoLinks
More VideoLinks >>>