Gene, Stem Cell Therapy Only Needs to be 50 Percent Effective to
Create a Healthy Heart, MU Researchers Find
Findings have major implications for those suffering from muscular
dystrophy and heart disease
Oct. 31, 2007
Story Contact: Christian Basi, (573) 882-4430, BasiC@missouri.
COLUMBIA, Mo. — 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.
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StemCells subscribers may also be interested in these sites:
Children's Neurobiological Solutions
http://www.CNSfoundation.org/
Cord Blood Registry
http://www.CordBlood.com/at.cgi?a=150123
The CNS Healing Group
http://groups.yahoo.com/group/CNS_Healing
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