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Sanford-Burnham Science Blog

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“Junk DNA” drives embryonic development

by Heather Buschman, Ph.D. on December 3, 2012 at 6:04 am | 0 Comments
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Differentiating mouse embryonic stem cells (green = mesoderm progenitor cells, red = endoderm progenitor cells). The microRNAs identified in this study block endoderm formation, while enhancing mesoderm formation.

Differentiating mouse embryonic stem cells (green = mesoderm progenitor cells, red = endoderm progenitor cells). The microRNAs identified in this study block endoderm formation, while enhancing mesoderm formation.

An embryo is an amazing thing. From just one initial cell, an entire living, breathing body emerges, full of working cells and organs. It comes as no surprise that embryonic development is a very carefully orchestrated process—everything has to fall into the right place at the right time. Developmental and cell biologists study this very thing, unraveling the molecular cues that determine how we become human.

“One of the first, and arguably most important, steps in development is the allocation of cells into three germ layers—ectoderm, mesoderm, and endoderm—that give rise to all tissues and organs in the body,” explains Mark Mercola, Ph.D., professor and director of Sanford-Burnham’s Muscle Development and Regeneration Program in the Sanford Children’s Health Research Center.

In a study published November 14 in the journal Genes & Development, Mercola and his team, including postdoctoral researcher Alexandre Colas, Ph.D., and Wesley McKeithan, discovered that microRNAs play an important role in this cell- and germ layer-directing process during development.

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#SBsymposium 2012: Stem cells & drug discovery

by Heather Buschman, Ph.D. on October 30, 2012 at 5:52 am | 0 Comments
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Symposium speakers Christine Mummery, Ph.D. (Leiden University Medical Center) and Mark Mercola, Ph.D. (Sanford-Burnham) both discussed their work on generating new heart muscle tissue from stem cells

Symposium speakers Christine Mummery, Ph.D. (Leiden University Medical Center) and Mark Mercola, Ph.D. (Sanford-Burnham) both discussed their work on generating new heart muscle tissue from stem cells

Scientists from all over San Diego—and beyond—gathered last Friday for Sanford-Burnham’s 34th annual symposium. This year’s theme: Frontiers in Stem Cell Biology for Drug Discovery. The topic was timely, given the recently announced 2012 Nobel Prize in Physiology or Medicine, awarded to John B. Gurdon and Shinya Yamanaka for their “discovery that mature cells can be reprogrammed to become pluripotent.” Yamanaka figured out how to turn a normal adult cell, such as a skin cell, into a stem cell that has the potential to become any other type of cell in the body. These special, laboratory-made stem cells are called induced pluripotent stem cells (iPSCs).

The symposium’s discussions centered on the idea that stem cells –especially iPSCs—can be used to model an individual’s own unique disease in a laboratory dish. These human cell-based models can then be used to test new and existing drugs for their toxicity and efficacy against disease.

Speakers came from Sanford-Burnham, Harvard, UT Southwestern, Mass General Hospital, UC San Diego, Stanford, and more. They talked about using stem cells to study and develop new therapies for conditions such as motor neuron disease, heart disease, autism, brain injury, Huntington’s disease, and spinal muscular atrophy.

We live-tweeted the event. For a snapshot of the day, including interesting tidbits, pictures, quotes, and links for more information, check out the Storify version of our tweets below. Then join the discussion on Twitter — look for us at @SanfordBurnham and #SBsymposium.

For more on stem cells and Sanford-Burnham’s work in the field, see:
Stem Cells 101
What is “Disease in a Dish”?
More stem cell blog posts
California Institute for Regenerative Medicine

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Stem cells 101

by Communications Staff on October 8, 2012 at 10:52 am | 2 Comments
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Sanford-Burnham's Stem Cell Research Center

Congratulations to John B. Gurdon and Shinya Yamanaka on winning the 2012 Nobel Prize in Physiology or Medicine! They received the award today for their “discovery that mature cells can be reprogrammed to become pluripotent.” In other words, these scientists figured out how to turn a normal adult cell, such as a skin cell, into a stem cell that has the potential to become any other type of cell in the body. Read below to learn more about stem cells and how they are revolutionizing medical research.

What are stem cells?

Stem cells are special because each is like a blank slate. Once it’s given the proper instruction, a stem cell can specialize and become any type of cell in the body—brain, heart, muscle, and more. Stem cells also have the ability to reproduce themselves indefinitely, renewing the supply.

Are there different types of stem cells?

Embryonic stem cells only exist during an organism’s development, when it is an embryo. These cells are pluripotent, meaning they have the capacity to become any cell type in the body.

Adult stem cells exist in fully developed organisms. They are more limited than embryonic stem cells—they are multipotent rather than pluripotent. These stem cells usually can only become a few types of specialized cells, based on the tissue from which they originate.

Induced pluripotent stem cells (iPSCs) are pluripotent, much like embryonic stem cells. iPSCs are produced in the laboratory by genetically reprogramming any adult cell, such as a skin cell.

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Long-term investments in research pay off

by Kristina Meek on September 26, 2012 at 10:43 am | 0 Comments
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Dr. Fred Levine worked tirelessly to discover a potential therapeutic for diabetes.

Dr. Fred Levine worked tirelessly to discover a potential therapeutic for diabetes.

Dr. Fred Levine, director of our Sanford Children’s Health Research Center, started searching for potential diabetes drugs in 2005. Back then, Sanford-Burnham didn’t have a high-throughput drug screening center. It didn’t even have a children’s health research center.

One day, Dr. Levine was conferring with his colleague Dr. Mark Mercola, a heart researcher. Dr. Mercola was using some modest drug screening equipment set up in a converted office down the hall from his laboratory. He was screening chemical compounds with the hope of finding a few they could further explore as potential drugs for treating heart disease. Dr. Levine thought the same technique might lead to potential treatments for type 1 (juvenile) diabetes.

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Mending a broken heart—with a molecule that turns stem cells into heart cells

by Heather Buschman, Ph.D. on August 2, 2012 at 9:01 am | 1 comment
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Stem cell-derived cardiomyocytes (heart muscle cells) expressing a green fluorescent protein

Stem cell-derived cardiomyocytes (heart muscle cells) expressing a green fluorescent protein

For years, scientists have been looking for a good source of heart cells that can be used to study cardiac function in the lab, or perhaps even to replace diseased or damaged tissue in heart disease patients. To do this, many are looking to stem cells. Researchers at Sanford-Burnham Medical Research Institute, the Human BioMolecular Research Institute, and ChemRegen, Inc. have been searching for molecules that convert stem cells to heart cells for about eight years—and now they’ve found one. Writing in the August 3 issue of Cell Stem Cell, the team describes how they sifted through a large collection of drug-like chemicals and uncovered ITD-1, a molecule that can be used to generate unlimited numbers of new heart cells from stem cells.

“Heart disease is the leading cause of death in this country. Because we can’t replace lost cardiac muscle, the condition irreversibly leads to a decline in heart function and ultimately death. The only way to effectively replace lost heart muscle cells—called cardiomyocytes—is to transplant the entire heart,” said Mark Mercola, Ph.D., director of Sanford-Burnham’s Muscle Development and Regeneration Program and senior author of the study. “Using a drug to create new heart muscle from stem cells would be far more appealing than heart transplantation.”

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New muscle research center opens in San Diego

by Heather Buschman, Ph.D. on January 27, 2012 at 6:23 am | 0 Comments
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The new San Diego Skeletal Muscle Research Center will be made up of three core facilities shared by five local institutions.

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

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.

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New therapeutic target for heart disease

by Heather Buschman, Ph.D. on November 17, 2011 at 11:34 am | 0 Comments
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Hyungsoo Kim, Ph.D., postdoctoral researcher and first author of the study

Mitochondria are often called cellular “powerhouses” because they convert nutrients into energy. But these tiny structures also help determine cellular lifespan. Scientists are now discovering how mitochondria alternate between duplicating and fragmenting and how these events help cells adapt to diverse physiological conditions.

In a paper published November 18 in Molecular Cell, a team led by Dr. Ze’ev Ronai discovered that the protein Siah2 regulates mitochondrial fragmentation under low oxygen conditions. The significance of these findings is demonstrated by the heart’s response to oxygen shortage and ischemia, the tissue damage caused by lack of oxygen, when the researchers inhibited Siah2. In cells and mice lacking the protein, heart cell death was prevented. As a result, tissue damage was reduced in a mouse model that mimics a heart attack.

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A few minutes with Mark Mercola

by Josh Baxt on May 2, 2011 at 12:00 pm | 2 Comments
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Dr. Mark Mercola

Dr. Mark Mercola

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…

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Leaders among peers

by Heather Buschman, Ph.D. on April 29, 2011 at 9:32 am | 0 Comments
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Wordle archive

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

by Amelia Tomas on April 21, 2011 at 3:54 pm | 1 comment
Full Article
Dr. Steven Smith, Scientific Director for the Florida Hospital Sanford-Burnham Translational Research Institute, demonstrates sophisticated equipment used in metabolic studies.

Dr. Steven Smith, Scientific Director for the Florida Hospital Sanford-Burnham Translational Research Institute, cares for a patient.

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.

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What is “Disease in a Dish?”

by Heather Buschman, Ph.D. on January 12, 2011 at 11:34 am | 6 Comments
Full Article
"Disease in a dish" has great potential to accelerate drug discovery.

"Disease in a dish" has great potential to accelerate drug discovery.

“Disease in a dish” is a cutting-edge, stem cell-based strategy that allows researchers to study an individual patient’s cells in a laboratory dish. Traditionally, scientists interested in a particular disease have used a standard cell line that has been grown in the lab for years or a mouse model (if one exists) that has been engineered to mimic the disease. Although extremely valuable, these techniques have obvious limitations. Animal models never entirely reflect the actual human condition – they don’t capture the complicated interplay between an individual patient’s genetics and the environmental factors that might influence the development of the disease or that patient’s response to a new therapy.

Read below to find out how diseases in a dish are made, how they’re being used to study and treat disease and how Sanford-Burnham researchers are applying the technique.

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