Forum for Science, Industry and Business

Sponsored by:     3M 
Search our Site:

 

U of M researchers find novel gene correction model for epidermolysis bullosa

07.06.2013
A research team led by pediatric blood and marrow transplantation experts Mark Osborn, Ph.D. and Jakub Tolar, M.D., Ph.D. from the Masonic Cancer Center, University of Minnesota, have discovered a remarkable new way to repair genetic defects in the skin cells of patients with the skin disease epidermolysis bullosa.

The findings, published today in the journal Molecular Therapy and highlighted in the most recent issue of Nature, represent the first time researchers been able to correct a disease-causing gene in its natural location in the human genome using engineered transcription activator-like effector nucleases.

Epidermolysis bullosa (EB) is a skin disease caused by genetic mutations. Patients suffering from EB – primarily children - lack the proteins that hold the epidermis and dermis together, which leads to painful blistering and sores. The condition is often deadly. The University of Minnesota is an international leader in the treatment of EB and the research that has led to new treatment approaches.

In their latest work, Osborn and Tolar's team collaborated with genomic engineer Daniel Voytas, Ph.D., of the University of Minnesota's College of Biological Sciences, to engineer transcription activator-like effector nucleases (TALENs) that target the mutation and correct the error in the skin cells of patients with the disease. Researchers then reprogrammed these cells to make pluripotent stem cells that can create many different kinds of tissues. These amended cells were then able to produce the missing protein when placed in living skin models.

"These results provide proof of principle for TALEN-based precision gene correction, and it could open the door for more individualized therapeutics," said Osborn, an assistant professor in the University of Minnesota Medical School's Department of Pediatrics Division of Blood and Marrow Transplantation.

By using an unbiased screening method, researchers were able to take a comprehensive approach to TALEN-mapping. This strategy helped identify three other possible locations for future research and potential therapies.

"This is the first time we've been able to seamlessly correct a disease-causing gene in its natural location in the human genome using the TALEN-based approach. This opened up options we did not have before when considering future therapies," said Tolar, director of the University's Stem Cell Institute and an associate professor in the Department of Pediatrics Division of Blood and Marrow Transplantation.

The University of Minnesota Pediatric Blood and Marrow Transplant team, led by John Wagner, M.D. and Bruce Blazar, M.D., has pioneered bone marrow transplantation as the standard of care for severe EB. Tolar and Osborn hope that the individualized "genome editing" of patient cells will provide the next generation of therapies for EB and other genetic diseases.

Funding for this research was supported by grants from the Epidermolysis Bullosa Research Fund, the Jackson Gabriel Silver Foundation, DebRA International, the University of Minnesota Academic Health Center, Pioneering Unique Cures for Kids Foundation, Children's Cancer Research Fund, and the United States of America Department of Defense. The National Institutes of Health supports several authors through grant R01 GM098861 and the Director's Pioneer Award DP1 OD006862.

Masonic Cancer Center, University of Minnesota is part of the University's Academic Health Center. It is designated by the National Cancer Institute as a Comprehensive Cancer Center. For more information about the Masonic Cancer Center, visit http://www.cancer.umn.edu or call 612-624-2620.

The University of Minnesota Medical School, with its two campuses in the Twin Cities and Duluth, is a leading educator of the next generation of physicians. Our graduates and the school's 3,800 faculty physicians and scientists advance patient care, discover biomedical research breakthroughs with more than $180 million in sponsored research annually, and enhance health through world-class patient care for the state of Minnesota and beyond. Visit http://www.med.umn.edu to learn more.

Caroline Marin | EurekAlert!
Further information:
http://www.umn.edu

More articles from Health and Medicine:

nachricht Purdue cancer identity technology makes it easier to find a tumor's 'address'
16.11.2018 | Purdue University

nachricht Microgel powder fights infection and helps wounds heal
14.11.2018 | Michigan Technological University

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: UNH scientists help provide first-ever views of elusive energy explosion

Researchers at the University of New Hampshire have captured a difficult-to-view singular event involving "magnetic reconnection"--the process by which sparse particles and energy around Earth collide producing a quick but mighty explosion--in the Earth's magnetotail, the magnetic environment that trails behind the planet.

Magnetic reconnection has remained a bit of a mystery to scientists. They know it exists and have documented the effects that the energy explosions can...

Im Focus: A Chip with Blood Vessels

Biochips have been developed at TU Wien (Vienna), on which tissue can be produced and examined. This allows supplying the tissue with different substances in a very controlled way.

Cultivating human cells in the Petri dish is not a big challenge today. Producing artificial tissue, however, permeated by fine blood vessels, is a much more...

Im Focus: A Leap Into Quantum Technology

Faster and secure data communication: This is the goal of a new joint project involving physicists from the University of Würzburg. The German Federal Ministry of Education and Research funds the project with 14.8 million euro.

In our digital world data security and secure communication are becoming more and more important. Quantum communication is a promising approach to achieve...

Im Focus: Research icebreaker Polarstern begins the Antarctic season

What does it look like below the ice shelf of the calved massive iceberg A68?

On Saturday, 10 November 2018, the research icebreaker Polarstern will leave its homeport of Bremerhaven, bound for Cape Town, South Africa.

Im Focus: Penn engineers develop ultrathin, ultralight 'nanocardboard'

When choosing materials to make something, trade-offs need to be made between a host of properties, such as thickness, stiffness and weight. Depending on the application in question, finding just the right balance is the difference between success and failure

Now, a team of Penn Engineers has demonstrated a new material they call "nanocardboard," an ultrathin equivalent of corrugated paper cardboard. A square...

All Focus news of the innovation-report >>>

Anzeige

Anzeige

VideoLinks
Industry & Economy
Event News

“3rd Conference on Laser Polishing – LaP 2018” Attracts International Experts and Users

09.11.2018 | Event News

On the brain’s ability to find the right direction

06.11.2018 | Event News

European Space Talks: Weltraumschrott – eine Gefahr für die Gesellschaft?

23.10.2018 | Event News

 
Latest News

Purdue cancer identity technology makes it easier to find a tumor's 'address'

16.11.2018 | Health and Medicine

Good preparation is half the digestion

16.11.2018 | Life Sciences

Microscope measures muscle weakness

16.11.2018 | Life Sciences

VideoLinks
Science & Research
Overview of more VideoLinks >>>