Stem cells for first time used to create abnormal heart cells for study of cardiomyopathy

Jun 09, 2010

Researchers at Mount Sinai School of Medicine have for the first time differentiated human stem cells to become heart cells with cardiomyopathy, a condition in which the heart muscle cells are abnormal. The discovery will allow scientists to learn how those heart cells become diseased and from there, they can begin developing drug therapies to stop the disease from occurring or progressing. The study is published in the June 9th issue of Nature.

The Mount Sinai team used from two patients with a known by the acronym LEOPARD syndrome. Hypertrophic , or thickening of the heart muscle, is experienced by 80 percent of patients with LEOPARD syndrome and is the most life-threatening aspect of the disorder. The Mount Sinai team took patient skin cells and reprogrammed them to become pluripotent . Such cells can then develop into almost any type of cell in the human body. The researchers then created heart cells that had characteristics of hypertrophic cardiomyopathy.

"We knew there was potential in using from people with genetic disorders to develop diseases in vitro, but our study is the first to successfully create abnormal heart cells," said the Principal Investigator of the study Ihor R. Lemischka, PhD, Professor, Gene and Cell Medicine, Developmental and Regenerative Biology, Mount Sinai School of Medicine. "Now that we have developed these cells, we can study why they become enlarged and develop treatments to prevent them from overgrowing."

Scientists know that genetic disorders occur because of a mutation in a protein signaling pathway called the RAS pathway, but they have been unable to determine precisely how this results in disease-associated problems like hypertrophic cardiomyopathy. The authors of the Nature study concluded that induced pluripotent stem cell-derived provide the required characteristics to precisely determine the pathology behind these disorders, and a foundation for studying treatment interventions.

"This discovery has broad-reaching implications for genetic diseases like LEOPARD syndrome and Noonan's syndrome," continued Dr. Lemischka. "We look forward to further studying these cells as a potential therapeutic target."

Explore further: DNA may have had humble beginnings as nutrient carrier

Provided by The Mount Sinai Hospital

3.8 /5 (4 votes)

Related Stories

Heart derived stem cells develop into heart muscle

Apr 23, 2008

Dutch researchers at University Medical Center Utrecht and the Hubrecht Institute have succeeded in growing large numbers of stem cells from adult human hearts into new heart muscle cells. A breakthrough in stem cell research. ...

Researchers make stem cell breakthrough

Mar 01, 2009

In a study to be released on March 1, 2009, Mount Sinai Hospital's Dr. Andras Nagy discovered a new method of creating stem cells that could lead to possible cures for devastating diseases including spinal ...

Stem cell regeneration repairs congenital heart defect

Sep 11, 2008

Mayo Clinic investigators have demonstrated that stem cells can be used to regenerate heart tissue to treat dilated cardiomyopathy, a congenital defect. Publication of the discovery was expedited by the editors of Stem Ce ...

Recommended for you

Research helps identify memory molecules

10 hours ago

A newly discovered method of identifying the creation of proteins in the body could lead to new insights into how learning and memories are impaired in Alzheimer's disease.

Computer simulations visualize ion flux

11 hours ago

Ion channels are involved in many physiological and pathophysiological processes throughout the human body. A young team of researchers led by pharmacologist Anna Stary-Weinzinger from the Department of Pharmacology ...

Neutron diffraction sheds light on photosynthesis

11 hours ago

Scientists from ILL and CEA-Grenoble have improved our understanding of the way plants evolved to take advantage of sunlight. Using cold neutron diffraction, they analysed the structure of thylakoid lipids found in plant ...

DNA may have had humble beginnings as nutrient carrier

Sep 01, 2014

New research intriguingly suggests that DNA, the genetic information carrier for humans and other complex life, might have had a rather humbler origin. In some microbes, a study shows, DNA pulls double duty ...

Central biobank for drug research

Sep 01, 2014

For the development of new drugs it is crucial to work with stem cells, as these allow scientists to study the effects of new active pharmaceutical ingredients. But it has always been difficult to derive ...

User comments : 0