Forum for Science, Industry and Business

Sponsored by:     3M 
Search our Site:

 

Cedars-Sinai, global consortium develop stem cell tool to test Huntington’s treatments

06.07.2012
From skin cells to brain cells, new “disease in a dish” model offers step forward in understanding fatal inherited disorder and ways to test therapies for it

Cedars-Sinai scientists, in collaboration with expert colleagues around the globe, used stem cells to re-create Huntington’s Disease in a petri dish, allowing researchers for the first time to test potential treatments for the fatal inherited neurological disorder directly on human cells.

As outlined in a paper published online by Cell Stem Cell and slated for print in the journal’s Aug. 3 issue, scientists at Cedars-Sinai’s Regenerative Medicine Institute and the University of Wisconsin took skin cells from patients with Huntington’s disease and reprogrammed them into powerful stem cells; these were then made into the nervous system cells affected by the disease. Seven laboratories around the world collaborated to demonstrate the cells had hallmarks of Huntington’s.

“Now that we’ve moved from skin cells to brain cells, the next step forward will be to test treatments we can move from the petri dish to the clinic,” said Clive Svendsen, PhD, director of the Cedars-Sinai Regenerative Medicine Institute and a senior author of the study. “In addition to increasing our understanding of this disorder and offering a new pathway to identifying treatments, this study is remarkable because of the extensive interactions between a large group of scientists focused on developing this model. It’s a new way of doing trailblazing science.”

This breakthrough is the latest example of an innovative use for stem cells, reprogramming them to create laboratory versions of diseases. This method has been used to mimic Parkinson’s disease, Alzheimer’s disease, ALS and spinal muscular atrophy. As these diseases all occur in living neurons – that patients can’t part with – the disease-in-a-dish models offer scientists an opportunity they never had before: to study in human cells how a disease attacks the neurons and plot strategies for fighting it.

The Huntington’s Disease iPSC Consortium united some of the world’s top scientists working on this disease. Cedars-Sinai researchers took skin cells from a several Huntington’s patients, including a six-year-old with a severe juvenile form of the disease. They genetically reprogrammed these tissues into induced pluripotent stem cells, which can be made into any type of cell in the body. The cells lines were banked by scientists at Cedars-Sinai and scrutinized by all consortium members for differences that may have led to the disease. These cell lines are now an important resource for Huntington’s researchers and have been made available via a National Institutes of Health-funded repository at Coriell Institute for Medical Research in New Jersey.

Huntington’s, known to the public, for example, as the cause of folksinger Woody Guthrie’s death, typically strikes patients in midlife. It causes jerky, twitching motions, loss of muscle control, psychiatric disorders and dementia; the disease ultimately is fatal. In rare, severe cases, the disorder appears in childhood.

Researchers believe that Huntington’s results from a mutation in the huntintin gene, leading to production of an abnormal protein and ultimately cell death in specific areas of the brain that control movement and cognition. There is no cure for Huntington’s, nor therapies to slow its progression.

The consortium showed Huntington’s cell deficits or how they differ from normal cells, including that they were less likely to survive cultivation in the petri dish. Scientists tried depriving them of a growth factor present around normal cells, or “stressing” them, and found that Huntington’s neurons died even faster. The trick with disease-in-a-dish models is verifying that the cells are, in fact, behaving in the same manner they would in a patient, said Virginia Mattis, a post-doctoral scientist at the Cedars-Sinai Regenerative Medicine Institute and one of the lead authors of the study.

“It was great that these characteristics were seen not only in our laboratory, but by all of the consortium members using different techniques,” Mattis said. “It was very reassuring and significantly strengthens the value of this study.”

This new model will provide the foundation for a new round of experiments by the consortium funded by a new grant from the NIH and the California Institute for Regenerative Medicine.

The Cedars-Sinai’s Regenerative Medicine Institute has made a major commitment to projects like this Huntington’s study in which stem cell research helps to advance understanding of human disease and open new and innovative methods to identify treatments and cures. The institute has developed an induced pluripotent stem cell core facility and recruited faculty to work in this emerging area of regenerative medicine research.

“At last, we have a human cell model for this tragic disease that will be a powerful new tool in identifying treatments for these patients,” said Shlomo Melmed, MD, dean of the medical faculty at Cedars-Sinai and the Helena A. and Philip E. Hixon Chair in Investigative Medicine. “This development is a compelling example of how important iPS cells are to furthering our understanding and finding cures for diseases that are currently untreatable.”

The consortium includes: Cedars-Sinai Medical Center; Johns Hopkins University School of Medicine in Baltimore; University of California, Irvine; University of Wisconsin School of Medicine; Massachusetts General Hospital; Harvard Medical School; University of California, San Francisco; Cardiff University; Universita degli Studi di Milano; and the CHDI Foundation.

Funding for the research came from an American Recovery and Reinvestment Act grant from the NIH’s National Institute of Neurological Disorders and Stroke (NINDS Recovery Act grant number: RC2-NS069422 ), a grant from the CHDI Foundation, Inc. and CIRM.

Nicole White | Cedars-Sinai News
Further information:
http://www.cshs.org

More articles from Life Sciences:

nachricht Colorectal cancer: Increased life expectancy thanks to individualised therapies
20.02.2020 | Christian-Albrechts-Universität zu Kiel

nachricht Sweet beaks: What Galapagos finches and marine bacteria have in common
20.02.2020 | Max-Planck-Institut für Marine Mikrobiologie

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 step towards controlling spin-dependent petahertz electronics by material defects

The operational speed of semiconductors in various electronic and optoelectronic devices is limited to several gigahertz (a billion oscillations per second). This constrains the upper limit of the operational speed of computing. Now researchers from the Max Planck Institute for the Structure and Dynamics of Matter in Hamburg, Germany, and the Indian Institute of Technology in Bombay have explained how these processes can be sped up through the use of light waves and defected solid materials.

Light waves perform several hundred trillion oscillations per second. Hence, it is natural to envision employing light oscillations to drive the electronic...

Im Focus: Freiburg researcher investigate the origins of surface texture

Most natural and artificial surfaces are rough: metals and even glasses that appear smooth to the naked eye can look like jagged mountain ranges under the microscope. There is currently no uniform theory about the origin of this roughness despite it being observed on all scales, from the atomic to the tectonic. Scientists suspect that the rough surface is formed by irreversible plastic deformation that occurs in many processes of mechanical machining of components such as milling.

Prof. Dr. Lars Pastewka from the Simulation group at the Department of Microsystems Engineering at the University of Freiburg and his team have simulated such...

Im Focus: Skyrmions like it hot: Spin structures are controllable even at high temperatures

Investigation of the temperature dependence of the skyrmion Hall effect reveals further insights into possible new data storage devices

The joint research project of Johannes Gutenberg University Mainz (JGU) and the Massachusetts Institute of Technology (MIT) that had previously demonstrated...

Im Focus: Making the internet more energy efficient through systemic optimization

Researchers at Chalmers University of Technology, Sweden, recently completed a 5-year research project looking at how to make fibre optic communications systems more energy efficient. Among their proposals are smart, error-correcting data chip circuits, which they refined to be 10 times less energy consumptive. The project has yielded several scientific articles, in publications including Nature Communications.

Streaming films and music, scrolling through social media, and using cloud-based storage services are everyday activities now.

Im Focus: New synthesis methods enhance 3D chemical space for drug discovery

After helping develop a new approach for organic synthesis -- carbon-hydrogen functionalization -- scientists at Emory University are now showing how this approach may apply to drug discovery. Nature Catalysis published their most recent work -- a streamlined process for making a three-dimensional scaffold of keen interest to the pharmaceutical industry.

"Our tools open up whole new chemical space for potential drug targets," says Huw Davies, Emory professor of organic chemistry and senior author of the paper.

All Focus news of the innovation-report >>>

Anzeige

Anzeige

VideoLinks
Industry & Economy
Event News

70th Lindau Nobel Laureate Meeting: Around 70 Laureates set to meet with young scientists from approx. 100 countries

12.02.2020 | Event News

11th Advanced Battery Power Conference, March 24-25, 2020 in Münster/Germany

16.01.2020 | Event News

Laser Colloquium Hydrogen LKH2: fast and reliable fuel cell manufacturing

15.01.2020 | Event News

 
Latest News

Active droplets

21.02.2020 | Medical Engineering

Finding new clues to brain cancer treatment

21.02.2020 | Health and Medicine

Beyond the brim, Sombrero Galaxy's halo suggests turbulent past

21.02.2020 | Physics and Astronomy

VideoLinks
Science & Research
Overview of more VideoLinks >>>