3/12/2014 12:19:42 PM
SAN DIEGO, Calif., March 12, 2014 ?? A team of cardiovascular researchers
from Sanford---Burnham Medical Research Institute (Sanford---Burnham), the
Cardiovascular Research Center at Icahn School of Medicine at Mount Sinai, and
the University of California, San Diego, have identified a small but powerful new
player in the onset and progression of heart failure. Their findings, published in
the journal Nature on March 12, also show how they successfully blocked the
newly discovered culprit to halt the debilitating and chronic life---threatening
condition in its tracks.
In the study, investigators identified a tiny piece of RNA called miR---25 that
blocks a gene known as SERCA2a, which regulates the flow of calcium in and
out of heart---muscle cells. Decreased SERCA2a activity is one of the main causes
of poor contraction of the heart and enlargement of heart---muscle cells leading to
Using a functional screening system developed by researchers at Sanford---
Burnham, the research team discovered miR---25 acts pathologically in patients
suffering from heart failure, delaying proper calcium uptake in heart---muscle
“Before the availability of high---throughput functional screening, our chance of
teasing apart complex biological processes involved in disease progression like
heart failure was like finding a needle in a haystack,” said study co---senior author
Mark Mercola, Ph.D., professor in the Development, Aging, and Regeneration
Program at Sanford---Burnham and professor of bioengineering at UC San Diego
Jacobs School of Engineering. “The results of this study validate our approach to
identifying microRNAs as potential therapeutic targets with significant clinical
Mercola’s laboratory has pioneered the use of robotic high---throughput methods
of drug discovery to identify new targets for heart failure. According to co---lead
study authors Christine Wahlquist and Agustin Rojas Muñoz, Ph.D., developers
of the approach and researchers in Mercola’s lab at Sanford---Burnham, they used
high---throughput robotics to sift through the entire genome for microRNAs
involved in heart---muscle dysfunction.
Subsequently, the researchers at the Cardiovascular Research Center at Icahn
School of Medicine at Mount Sinai found that injecting a small piece of RNA to
inhibit the effects of miR---25 dramatically halted heart---failure progression in mice.
In addition, it also improved their cardiac function and survival.
“In this study, we have not only identified one of the key cellular processes
leading to heart failure, but have also demonstrated the therapeutic potential of
blocking this process,” said co---lead study author Dongtak Jeong, Ph.D., a post---
doctoral fellow at the Cardiovascular Research Center at Icahn School of
Medicine at Mount Sinai in the laboratory of the study’s co---senior author Roger J.
Nearly six million Americans suffer from heart failure, which is when the heart
becomes weak and cannot pump enough blood and oxygen throughout the body.
Heart failure is a leading cause of hospitalization in the elderly. Often, a variety
of medications are used to provide heart---failure patients temporary relief of their
debilitating symptoms. However, these medications do not improve cardiac
function or halt the progression of the disease.
“Currently, heart---failure medications do not effectively address the underlying
mechanisms that weaken contractile function and lead to the enlargement of
heart---muscle cells,” said co---senior study author Roger J. Hajjar, M.D. “Our study
provides us with the key evidence we need to begin developing miR---25 as an
important new therapeutic target, while adding our successful technique to block
this microRNA to our growing arsenal of promising heart---failure therapies that
we will further develop and test in future clinical trials.”
Currently, Hajjar’s laboratory is developing novel gene therapies for patients
with heart failure. One therapy, currently in phase IIb/III human clinical trials,
uses a modified viral vector to deliver a gene that produces SERCA2a, an
enzyme found in healthy heart---muscle cells. Another therapy in preclinical
development uses a disabled virus to deliver a gene called SUMO---1, which
shrinks enlarged heart---muscle cells and improves cardiac function.
About Sanford---Burnham Medical Research Institute
Sanford---Burnham Medical Research Institute is dedicated to discovering the
fundamental molecular causes of diseases and devising the innovative therapies
of tomorrow. Sanford---Burnham takes a collaborative approach to medical
research with major programs in cancer, neurodegeneration, diabetes, and
infectious, inflammatory and childhood diseases. The Institute is recognized for
its National Cancer Institute---designated Cancer Center and expertise in drug
discovery and stem---cell technologies. Sanford---Burnham is a nonprofit,
independent institute that employs 1,200 scientists and staff in San Diego (La
Jolla), Calif., and Orlando (Lake Nona), Fla. For more information, visit us at
About the Mount Sinai Health System
The Mount Sinai Health System is an integrated health system committed to
providing distinguished care, conducting transformative research, and
advancing biomedical education. Structured around seven member hospital
campuses and a single medical school, the Health System has an extensive
ambulatory network and a range of inpatient and outpatient services—from
community---based facilities to tertiary and quaternary care.
The System includes approximately 6,600 primary and specialty care physicians,
12 minority---owned free---standing ambulatory surgery centers, over 45
ambulatory practices throughout the five boroughs of New York City,
Westchester, and Long Island, as well as 31 affiliated community health centers.
Physicians are affiliated with the Icahn School of Medicine at Mount Sinai, which
is ranked among the top 20 medical schools both in National Institutes of Health
funding and by U.S. News & World Report. For more information, visit
Find Mount Sinai on:
Twitter: @mountsinainyc http://www.twitter.com/mountsinainyc
About the UC San Diego Jacobs School of Engineering
The UC San Diego Jacobs School of Engineering is a premier research
organization dedicated to educating tomorrow’s technology leaders and
conducting leading edge research that drives innovation for the benefit of society.
The Jacobs School’s Department of Bioengineering is ranked in the top 3 of its
kind in the nation, according to various rankings. The bioengineering
department is a world leader, focused on the understanding, diagnosis and
treatment of human disease through bioengineering analysis, systems biology
and regenerative medicine.
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