Top Stories - Muscle Development & Regeneration

The new San Diego Skeletal Muscle Research Center will be made up of three core facilities shared by five local institutions.
New muscle research center...

The NIH recently awarded a new grant to establish the San Diego Skeletal Muscle Research Center,...

Lorenzo Puri, M.D., Ph.D.
Muscling up with MyoD

Lorenzo Puri and his lab members study what makes stem cells choose to produce the proteins that...

Dr. Mark Mercola
A few minutes with Mark...

Dr. Mark Mercola talks about cardiomyocyte regeneration and protection research and the June 20...

Dr. Alessandra Sacco
New mouse model for muscular...

Roughly 50,000 people in the United States are affected by some type of muscular dystrophy, a...

New muscle research center opens in San Diego

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The National Institutes of Health (NIH) recently awarded a new grant to establish the San Diego Skeletal Muscle Research Center. This new center, led by UC San Diego’s Rick Lieber, Ph.D., Sanford-Burnham’s Mark Mercola, Ph.D., and The Scripps Research Institute’s Velia Fowler, Ph.D., will allow 21 scientists at five different research institutions to combine their expertise and state-of-the-art methods to accelerate  research that advances our understanding of skeletal muscles and the diseases that affect them.

Muscling up with MyoD

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Every cell in your body contains the same DNA, with genes coding for many thousands of proteins. Yet a muscle cell makes a very different set of proteins from say, a bone cell, enabling it to perform its muscle-specific job. Lorenzo Puri, M.D., Ph.D. and his lab members study what makes stem cells (precursor cells) choose to produce the proteins that turn them into muscle cells. In doing so, they hope their research will one day help improve strategies for muscle regeneration in patients with muscle wasting diseases, such as muscular dystrophy. While a cure for muscular dystrophy is not yet in sight, the ability to stimulate muscle stem cells to generate mature muscle cells could make a big difference in the lives of patients. By repairing muscles damaged by the disease, muscle regeneration therapy would extend the lives of patients and allow them to function effectively in a whole range of activities that are currently unthinkable for victims of the disease.

A few minutes with Mark Mercola

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Dr. Mark Mercola directs Sanford-Burnham’s Muscle Development and Regeneration Program and is looking for ways to regenerate damaged heart tissue. He is particularly interested in growing cardiomyocytes (beating heart cells) from stem cells and finding ways to spur a person’s existing heart precursor cells, which can already heal small injuries, to work harder to tackle major heart disease.

On June 20-21, 2011, Dr. Mercola is chairing a conference on Cardiomyocyte Regeneration and Protection. Sponsored by Abcam, the conference will combine basic and clinical research findings to move us closer to new treatments. Recently, Dr. Mercola talked to Abcam about the upcoming conference…

Leaders among peers

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Sanford-Burnham scientists are leading several exciting symposia over the next few months. Please follow the links below for more event and registration information.

2011 Signaling, Metabolism and Hypoxia Symposium
Chaired by Dr. Ze’ev Ronai

May 6, 2011, 2:00 – 5:30 p.m. (PDT)
Sanford-Burnham Medical Research Institute
10901 North Torrey Pines Road
La Jolla, California

2011 Glycobiology Gordon Research Conference
Chaired by Dr. Hudson Freeze

May 8 – 13, 2011
Il Ciocco Hotel
Lucca (Barga), Italy

Sanford-Burnham’s 33rd Annual Symposium: Structural Systems Biology
Chaired by members of the Bioinformatics and Systems Biology Program
Drs. Adam Godzik, Dorit Hanein, Andrei Osterman, Niels Volkmann

June 7, 2011, 9:00 a.m. – 5:15 p.m. (PDT)
Hilton La Jolla Torrey Pines
La Jolla, California

Cardiomyocyte Regeneration and Protection
Chaired by Dr. Mark Mercola

Sponsored by Abcam
June 20 – 21, 2011
Hilton La Jolla Torrey Pines
La Jolla, California

2011 Molecular Therapeutics of Cancer Research Conference
Chaired by Dr. Sara Courtneidge

Sponsored by the Cancer Molecular Therapeutics Research Association
July 10 – 14, 2011
Asilomar Conference Center
Pacific Grove, California

Seventh General Meeting of the International Proteolysis Society
Chaired by Dr. Guy Salvesen and Dr. Matthew Bogyo

October 16 – 20, 2011
Hilton San Diego Resort and Spa
San Diego, California

Personalized Medicine 101

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In 2003, the completion of the human genome project gave us an unprecedented amount of genetic information. From this, a new clinical concept is emerging: personalized medicine.Conventional medical care generalizes treatment to all patients with a particular disease. But since a disease is as individual as the person who has it, casting a wide therapeutic net has its limitations. For one, patients with a certain genetic makeup might not respond to a particular drug as well as patients with different genetics, or they might experience different side effects. As personalized medicine becomes a reality, it could rectify these less-than-ideal situations.

From the diagnostic point-of-view, personalized medicine is a shift from reactive to proactive. Based on a person’s health, genetic, and environmental profiles, doctors practicing personalized medicine could assess a patient’s risk for acquiring a genetic disease before any symptoms develop. This might allow them to target the specific genes that account for illness (the BRCA1/BRCA2 genes that predispose a woman to breast cancer, for example), incorporate a prevention strategy, and monitor those genes over time. When it comes to treatment, personalized drugs could be prescribed based on an individual’s molecular “build” and targeting treatment where it will do the most good and the least harm.

New mouse model for muscular dystrophy

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Roughly 50,000 people in the United States are affected by some type of muscular dystrophy, a condition characterized by debilitating muscle loss. Duchenne muscular dystrophy (DMD), the most common form of the disease, is caused by a mutation in the dystrophin gene. Without dystrophin, the interior muscle fiber frame can’t properly connect to the surrounding environment. Despite this knowledge, lack of a good DMD model has long hampered scientists in their efforts to study the disease and develop new therapies. Although the current DMD mouse model replicates the dystrophin gene mutation seen in humans, the mice don’t suffer the same devastating symptoms. Writing December 9 in the journal Cell, researchers in Dr. Helen Blau’s group at Stanford University, including Drs. Alessandra Sacco, Foteini Mourkioti and Jason Pomerantz, uncover the molecular reason for this model’s shortcomings, and use that information to create a more accurate mouse model for DMD.

Dr. Sacco, now assistant professor at Sanford-Burnham, notes the importance of this new mouse model. “There are no effective cures for DMD. Several therapies seemed promising after they cured muscular defects in mice, but they failed in human clinical trials. Our new model will provide a more precise tool for studying muscular dystrophy and testing new therapies.”

Putting the muscle in muscle stem cells

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Patients with muscular dystrophy suffer debilitating muscle loss that gets worse as they age. As the disease progresses, resident stem cells in a patient’s muscle tissue have to work extra hard trying to replace the diseased muscle. Over time, this special population of stem cells gets exhausted as they constantly proliferate (make more stem cells) and differentiate (specialize into new muscle cells).

Dr. Pier Lorenzo Puri, associate professor at Sanford-Burnham and Italy’s Dulbecco Telethon Institute, and colleagues are figuring out ways to keep the muscle stem cell pool fresh and ready to regenerate injured or diseased muscle. In a study published today in the journal Cell Stem Cell, they uncover the molecular messengers that translate inflammatory signals into the genetic changes that tell muscle stem cells to differentiate. These findings give the scientists a target to artificially dial the stem cell population up or down, a potential treatment that could boost muscle regeneration in muscular dystrophy patients.

“Our mission is to improve the lives of these patients and extend their lives until they can benefit from a cure 20 years from now,” says Dr. Puri,  a medical doctor who has worked with many muscular dystrophy patients throughout his career.