Gene, stem cell therapy only needs to be 50 percent effective to create a healthy heart

October 31, 2007

Heart disease is the leading cause of death in the United States and greatly affects the quality and length of life for individuals with specific forms of muscular dystrophy. Recent discoveries have demonstrated that gene and/or stem cell therapy could help a variety of organs in the body, but until now scientists have been unsure whether the heart could benefit from these treatments.

According to a new study, recently published in Circulation Research, a journal of the American Heart Association, University of Missouri-Columbia researchers have demonstrated that a muscular dystrophy patient should be able to maintain a normal lifestyle if only 50 percent of the cells of the heart are healthy.

Patients with Duchenne muscular dystrophy and Becker muscular dystrophy have a gene mutation that disrupts the production of a protein known as dystrophin. Absence of this protein starts a chain reaction that eventually leads to muscle cell degeneration and death. Eventually, the damaged muscle tissue is replaced by fibrous, bony or fatty tissue and loses function. In the heart, this leads to severe heart disease and can place severe limitations on individuals afflicted with the disease.

In the past, scientists believed that the only way to have a healthy heart was to rid the heart of all damaged tissue. The heart is considered to be a “synchronized organ;” therefore, it was believed that the heart needed to maintain 100 percent normal cells in order to stay healthy.

In gene therapy, mutated genes are replaced with healthy genes. In stem cell therapy, diseased cells are replaced with healthy cells. However, in these gene and stem cell therapies, it is not feasible to fix every cell in the heart. Previously, scientists were uncertain whether partial correction could benefit patients.

“In our study, we found that a heart with 50 percent normal cells looks like a normal heart,” said Dongsheng Duan, an associate professor of molecular microbiology and immunology at the MU School of Medicine. “More importantly, it acts like a normal heart. This is the first time that we have concrete evidence that partial gene or cell therapies will be effective for preventing heart disease in a mouse model of muscular dystrophy.”

“It’s important to note that this could improve the quality of life for individuals who have this heart condition,” said Brian Bostick, a doctoral student in molecular microbiology and immunology and the first author of the study. “We’re also looking at this as a possible way to prevent heart disease. If we can treat it early through gene therapy or cell therapy, we know now that it can be very beneficial for patients.”

The MU researchers said that this finding would have a positive impact on the ongoing gene and cell therapy studies in animal models of muscular dystrophy as well as in human patients. It also raises the hope of developing effective gene and cell therapies for patients suffering from other heart diseases.

Source: University of Missouri-Columbia

Explore further: A change of heart: Epigenetic basis of cardiac hypertrophy uncovered

Related Stories

Team releases gene edited human stem cell lines

November 30, 2016

The Allen Institute for Cell Science has released the Allen Cell Collection: the first publicly available collection of gene edited, fluorescently tagged human induced pluripotent stem cells that target key cellular structures ...

Scientists create heart cells better, faster, stronger

November 10, 2016

Scientists at the Gladstone Institutes identified two chemicals that improve their ability to transform scar tissue in a heart into healthy, beating heart muscle. The new discovery advances efforts to find new and effective ...

Recommended for you

Artificial beta cells

December 8, 2016

Researchers led by ETH Professor Martin Fussenegger at the Department of Biosystems Science and Engineering (D-BSSE) in Basel have produced artificial beta cells using a straightforward engineering approach.

Key regulator of bone development identified

December 8, 2016

Loss of a key protein leads to defects in skeletal development including reduced bone density and a shortening of the fingers and toes—a condition known as brachydactyly. The discovery was made by researchers at Penn State ...

Researchers question lifelong immunity to toxoplasmosis

December 8, 2016

Medical students are taught that once infected with Toxoplasma gondii—the "cat parasite"—then you're protected from reinfection for the rest of your life. This dogma should be questioned, argue researchers in an Opinion ...

TET proteins drive early neurogenesis

December 7, 2016

The fate of stem cells is determined by series of choices that sequentially narrow their available options until stem cells' offspring have found their station and purpose in the body. Their decisions are guided in part by ...

0 comments

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.