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The Heart of Research T. Denny Sanford Does it Again Leadership Transitions




A Message from John Reed, M.D., Ph.D. 1

Dr. William H. and Lillian Fishman

A Message from Kristiina Vuori, M.D., Ph.D. 2


Research is Vital 4

Roberta and Malin Burnham Joe Lewis Conrad T. Prebys T. Denny Sanford

Thank you to T. Denny Sanford 3 Philanthropy is Vital 5 The Heart of Research 6 Talking with a Donor: Charles “Skeets” Dunn 8


Talking with a Scientist: Dwight Towler, M.D., Ph.D. 9

M. Wainwright Fishburn, Jr.

Recreating a Disease in a Dish 10




Upcoming Events 11 Recent Events 12 Partners in Science Back Cover



Gary F. Raisl, M.B.A., Ed.D.

Deborah Robison





Margaret M. Dunbar, J.D.

Paul Baker Stephanie Boumediene, M.P.H. Philip Graham, M.B.A.


Lorenzo Berho James C. Blair Shehan Dissanayake, Ph.D. Daniel J. Epstein Pauline M. Foster Patrick J. Geraghty Alan A. Gleicher Jeanne L. Herberger, Ph.D. Brent Jacobs James E. Jardon II Daniel Kelly, M.D. Robert J. Lauer J. Bernard Machen, D.D.S., Ph.D. Hank Nordhoff Douglas Obenshain Peter Preuss Andrew J. Viterbi, Ph.D. Allen R. Weiss Gayle E. Wilson EX-OFFICIO



Edgar M. Gillenwaters


Kristina Meek


Heather Buschman, Ph.D.

The Heart of Research T. Denny Sanford Does it Again Leadership Transitions



Creative Fusion


Researchers work together each day to solve problems in Sanford-Burnham laboratories, like Dr. Masa Komatsu’s. Our donors take a strong interest in understanding our work, as T. Denny Sanford did when he visited Dr. Fred Levine’s lab.

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A Message from

Dr. John Reed It has been my honor and privilege to lead Sanford-Burnham for the past 11 years in the role of CEO, and for 21 years as a member of the faculty in various leadership positions. During that time, the Institute has emerged as one of the world’s leading biomedical research institutes thanks, in part, to the generosity of our donors. All organizations benefit from fresh leadership approximately every decade, and that time is now for Sanford-Burnham. I have decided to step down as CEO in order to accept the position as Global Head of Pharma Research and Early Development at Roche, and member of the company’s executive committee. I will maintain an adjunct professor position with the Institute and look forward to continued interactions with our scientific teams in the years ahead. Dr. Kristiina Vuori has taken the reins as interim CEO. Many of you are already familiar with her bold leadership and scientific brilliance. I am confident that a bright future lies ahead for Sanford-Burnham. The Institute will always will be a significant chapter in my career. Thank you for helping make it a memorable one.

Sincerely, John C. Reed, M.D., Ph.D. | PORTAL


A Message from

Dr. Kristiina Vuori It is with pride and gratitude that I assume the role of interim chief executive officer of Sanford-Burnham. During the 20-plus years that I have been with the organization , I have been amazed to witness Sanford-Burnham’s growth. The Institute has become a world leader in medical research and early-stage drug discovery, and I am confident that we will continue on that trajectory. I hope that you feel the same pride that I do in our accomplishments. I will continue to serve as president of the Institute and, for the near future, lead our National Cancer Institute-designated Cancer Center, the largest research center at Sanford-Burnham. As a research scientist, I also oversee a talented team that is seeking to halt metastasis and make cancer a more easily treatable disease. My commitment to this work continues uninterrupted. The Institute is positioned at the forefront of basic research and drug discovery, equipped with the very best talent and technology. Please join me in continuing Sanford-Burnham’s forward momentum.




We’re happy to announce that top cancer expert, Garth Powis, Ph.D., has been appointed professor and director of our Cancer Center, one of seven National Cancer Institute (NCI)-designated basic research cancer centers in the U.S. He will also assume the Jeanne and Gary Herberger Leadership Chair in Cancer Research. Dr. Powis previously held leadership positions at MD Anderson Cancer Center in Texas. He’ll join our faculty May 1. We’ll tell you more about Dr. Powis and his vision for the Cancer Center in our June issue.

Thank You to T. Denny Sanford In February, philanthropist T. Denny Sanford reaffirmed his commitment to Sanford-Burnham and expressed his confidence in our interim chief executive officer, Kristiina Vuori, M.D., Ph.D., by pledging a substantial donation for cancer research. “At this time of transition, I want to provide both financial support and a personal endorsement of Sanford-Burnham’s excellence,” said Mr. Sanford. “I have the utmost confidence in the Institute’s future and Dr. Vuori’s leadership as interim CEO.” Mr. Sanford’s previous pledges to the Institute total in excess of $70 million, including the transformative gift that resulted in the Institute’s name change in 2010. “Mr. Sanford has made an indelible mark on this institution through financial support, which he has characterized as investments in medical research. He envisions a healthier future and we are honored to be part of that vision,” Dr. Vuori said. “As a cancer researcher, this latest gift means a great deal to me personally, as well as to the Institute.” Dr. Vuori and her research team are investigating how individual tumors can differ genetically and why a treatment that works for one patient may not work for another. To find therapeutic drugs that work for specific breast tumor types, her team will work with drug discovery experts in our Conrad Prebys Center for Chemical Genomics. The goal is to identify chemical compounds that have the potential to become new, more personalized, breast cancer therapies. South Dakota-based Sanford Health, also supported by Mr. Sanford and home to the Edith Sanford Breast Cancer Foundation, shares a commitment to unlocking the full potential of genomics to eradicate breast cancer, and will participate in the study. A genomics-based approach is one of the most promising ways of treating certain cancers. With investments from philanthropists like T. Denny Sanford, Sanford-Burnham researchers will leverage the Institute’s powerful technology platforms to achieve continued success in drug discovery for cancer and numerous other diseases. | PORTAL


Research is Vital When you take a pill or see your doctor for a routine test, do you think about how that pill or test came into existence? It almost certainly started in a laboratory. Chances are it grew from a combination of many different people’s ideas, all of which had to endure extensive testing. It took time. Here’s an example. Sir Alexander Fleming first discovered that the mold Penicillium notatum had an antibacterial agent in 1928. It wasn’t until 20 years later that Andrew J. Moyer patented a method of mass production of penicillin. At the time, it was the only weapon against certain deadly infections, and today it is the most widely used antibiotic. Who knows how many lives might be saved 20 years from now by a discovery made in the lab today? That discovery may just need a push to start it on its path to the drug pipeline. It may take a brilliant idea on the part of a scientist, or the right technology, or just a new way of looking at it. It’s vital that scientists keep striving each day to move each small discovery toward its clinical potential, and Sanford-Burnham provides the environment to do that. Last year we told you about sabutoclax, a potential cancer drug that originated from research by Sanford-Burnham’s Maurizio Pellecchia, Ph.D., and John C. Reed, M.D., Ph.D. At the time, biotechnology company Oncothyreon had just begun developing it. Now, sabutoclax has taken another important step toward saving lives. A study published in January shows its potential, in combination with other therapies, to treat chronic myeloid leukemia (CML) in the laboratory. (Learn more on our blog at beaker. Countless other potential medicines await discovery and development. Our scientists are making progress every day, and we hope that you’re as excited as we are to see what the future holds.

“We live in a time of unprecedented scientific opportunities biomedical science.” – from the American Association fo 4


Philanthropy is Vital “Philanthropy is critically important,” says Robert WechslerReya, Ph.D., professor and director of Sanford-Burnham’s Tumor Development Program. Dr. Wechsler-Reya recently gave an interview in which he addressed the importance of philanthropic support for medical research, particularly in the promising field of personalized medicine. Dr. Wechsler-Reya was the first to identify a new type of stem cell that can develop mutations and thus give rise to medulloblastoma, the most common malignant brain cancer in children. His team in our National Cancer Institute-designated Cancer Center is also creating laboratory models that can be used to test new drugs on this devastating cancer. “We have to look at each patient’s cancer as a unique disease,” he says. “If we had more money for research, we’d be able to make personalized medicine a reality. A cancer patient would go in for surgery and a piece of his or her tumor would be transported to the lab. In the lab, we’d test a battery of candidate drugs on the tumor. Then we could report to the clinic which drugs worked and which didn’t. Physicians would then use this information to make more individualized therapy decisions. “That sounds simple, but each step is complicated and takes careful coordination. Most importantly, the approach requires validation—we need to do this enough times to know that what works on a tumor in the laboratory is also likely to work in the patient. All this takes money and manpower.” He went on to say that, historically, the National Institutes of Health has supported cancer research in this country, but such funding is in decline. He explains, “As a result, research institutions are becoming increasingly dependent on philanthropy and individual contributions. Donations can really make a big difference in transforming a discovery into a new treatment.”

s, afforded to us by past investments in cancer research and or Cancer Research, in their 2012 Cancer Progress Report | PORTAL


The Heart of Research There’s a reason we use the term “at the heart of” to describe something that is integral to a system. Your heart is connected, directly or indirectly, to just about every other part of your body. Many aspects of your health depend on it. In the same way, heart research connects with—and overlaps with—numerous other areas of human biology. Sanford-Burnham fosters an environment where those connections can flourish. Diabetes and Obesity Research Center Sanford-Burnham’s Cardiovascular Pathobiology Program resides within the Diabetes and Obesity Research Center at our Lake Nona, Orlando, campus. Heart health is intricately linked with diabetes and obesity, so the programs at that location fit naturally together. Daniel Kelly, M.D., scientific director at Lake Nona, says, “The more we study diabetes, obesity, metabolism and heart health, the more overlap we discover. Each new finding in any one area seems to unlock potential new findings in another. We’re very fortunate to have such top-notch scientists working in proximity, so that we can learn from each other’s research.” Dwight Towler, M.D., Ph.D., whom you can learn more about on page 9, is a new faculty member who brings the Institute extensive knowledge of the devastating effect type 2 diabetes can have on the cardiovascular system. He studies the molecular causes of arterial calcification, or hardening of the arteries. This condition is a common complication in type 2 diabetes, where it can sometimes necessitate lower extremity amputation. “Dr. Towler’s research will be instrumental in our quest to find novel treatments for diabetes and obesity,” said Dr. Kelly. Dr. Towler’s research dovetails with the work of Masanobu Komatsu, M.D., Ph. D., whose interests encompass cardiovascular disease and cancer. Like Dr. Towler, Dr. Komatsu studies blood vessels, but from a different angle. Abnormal growth



and malfunction of blood vessels plays a role in many medical conditions, including heart disease and tumor development. Dr. Komatsu and his team are looking for ways to use blood vessels to enhance cancer drug delivery, avoid blocked arteries, and slow eyesight deterioration in elderly people. Research on Both Coasts Heart disease research isn’t limited to the one center at the Lake Nona campus—or limited even to heart disease specialists. Ze’ev Ronai, Ph.D., scientific director at our La Jolla campus and associate director of our National Cancer Institute-designated Cancer Center, devotes much of his resources to studying melanoma and other cancers. However, he made an important finding about how a particular protein regulates mitochondria, the energy-producing powerhouses of a cell. The finding relates to how lack of oxygen during a heart attack leads to heart tissue damage. Thus, his team has identified a potential new target for heart disease therapies. For many years, Erkki Ruoslahti, M.D., Ph.D., distinguished professor in the Cancer Center, has been using specially designed peptides (short proteins) to target tumors. While pursuing this research, his lab discovered a peptide that can guide drugs or imaging agents specifically to plaque in the arteries. These are just two examples of how research focused on another disease area led to breakthroughs for cardiovascular health. Heart Disease and Stem Cells Recently, there have been many developments in stem cell research as it relates to heart disease. This is thanks, in part, to SanfordBurnham’s Stem Cell Research Center, a resource to all researchers in both La Jolla and Lake Nona. The California Institute for Regenerative Medicine, the state’s stem cell funding agency, awarded a $1.58 million grant to associate professor Huei-Sheng Vincent Chen, M.D., Ph.D., to study an inherited form of heart disease. He and his team are creating personalized models of the disease using

Cardiovascular complications are the number one cause of death in diabetics. laboratory-made stem cells. (Learn more on page 10.) Scientists have long sought a good source of heart cells that can be used to replace diseased or damaged tissue in heart disease patients. Mark Mercola, Ph.D., director of our Muscle Development and Regeneration Program, found one. His team sifted through a large collection of drug-like chemicals and uncovered ITD-1. This molecule can be used to generate unlimited numbers of new heart cells from stem cells. It could become the basis for a new therapeutic drug that would limit scar spreading in heart failure and promote new muscle formation. Bolstered by this success, Dr. Mercola and collaborators founded ChemRegen, Inc., a spin-out company that aims to bring ITD-1 and other molecules like it to the clinic, where they could help heal patients in need. It’s all Connected With all of these researchers working together, sharing knowledge and resources, discoveries are never limited to one disease or one organ of the body. Cardiovascular research is just one example of a vast field that interconnects with many others. Whatever our researchers learn, you can bet that they will leverage that knowledge to its fullest potential, striving to treat and cure diseases of all kinds.

HEARTS IN SPACE Later this year, a team led by Rolf Bodmer, Ph.D., Karen Ocorr, Ph.D., and collaborators will send fruit flies to the International Space Station. These tiny organisms are ideal for modeling human heart health. Ultimately, the work could lead to countermeasures to prevent or treat heart problems—both in space and on Earth. | PORTAL



Charles “Skeets” Dunn, pictured with a Ferrari 212 Vignale, his personal favorite among his entire collection.

Charles “Skeets” Dunn:

The Finer Things

Charles “Skeets” Dunn knows quality when he sees it. His Ferrari collection is one of the finest in Southern California, as guests discovered at an exclusive event that he and his wife, Sharon, hosted in February. More importantly, though, he seeks out quality nonprofit organizations in which to invest his wealth. “One hundred percent of our money is going to charity,” Dunn says. “All of the organizations are ones I know will put it to good use.” Sanford-Burnham is honored to be included among those. Dunn surprised and delighted the guests at our 2012 fundraising gala when he announced that he was bequeathing $500,000 to the Institute. Dunn has enjoyed getting to know SanfordBurnham professor John Reed, M.D., Ph.D., who, until recently, also served as the Institute’s CEO. The two of them often talk about the promising future of research, particularly in the area of genomics. “What’s exciting to me,” Dunn says, “is all that Sanford-Burnham



is doing with the genome, and how you can target specific genes with specific treatments.” A highly accomplished retired bond trader and former Air Force service member, Dunn has been familiar with Sanford-Burnham for many years. He first learned about the Institute through Malin Burnham, San Diego community leader and one of our earliest supporters. Dunn viewed Burnham’s involvement as a stamp of excellence. He recalls a more recent conversation with Burnham: “I asked him, ‘What is most important to you’? and he said, unequivocally, ‘The Institute.’” Dunn jokes about the details of medical research being “over his head.” It’s true that the language of scientific research can sound foreign to a non-scientist. But, he points out, one doesn’t have to be a scientist to know that research is necessary to tackle major problems facing our society, like rising diabetes and obesity rates. “I really am a neophyte when it comes to medicine, but I’ve been very moved by the things they’re doing … by the energy and the avant garde nature of the Institute,” he says. Another of Dunn’s passions is his alma mater, Franklin & Marshall College in Lancaster, Pa. He started a scholarship fund there 30 years ago, and Sanford-Burnham recently played a role in the success of one scholar. The young woman was unable to afford medical school immediately after completing her undergraduate work. So she took a year off to work. “She’d always dreamed about being in San Diego and being at a research institute,” Dunn says. He helped arrange for her to work in the lab of Evan Snyder, M.D., Ph.D., director of our Stem Cell and Regenerative Biology Program. Following that experience, she was accepted to Harvard Medical School with a full scholarship. At Sanford-Burnham, we are always honored and inspired by the enthusiastic support of our donors. “Skeets” Dunn is no exception. “I’m thrilled by the things the Institute is doing,” he concludes. “It’s a phenomenal organization. It’s going to save people’s lives.”


Dwight Towler:

Learning From Patients “You don’t have to look far to find someone who has had an aortic valve replacement,” says Dwight Towler, M.D., Ph.D. He names Barbara Bush and Arnold Schwarzenegger as two famous examples. If you or someone you know has had heart surgery, you’re probably aware of the need for better treatments. So is Dr. Towler. He is a world-renowned endocrinologist and vascular biologist making great strides in the lab—but it’s his previous work with patients that drives him. In November, Dr. Towler became professor and director of Sanford-Burnham’s Cardiovascular Pathobiology Program, based at the Lake Nona campus in Orlando. He is excited to be a part of the blossoming life-science hub in Lake Nona known as Medical City. “It’s very interesting to see this center of science and medicine as it’s being built from the ground up,” he says. Dr. Towler studies the molecular causes of arterial calcification, or hardening of the arteries. This condition is a common complication in type 2 diabetes and chronic kidney disease, where it can sometimes lead to lower extremity amputation. Arterial calcification also increases risk for stroke and heart attack. Board-certified in internal medicine, Dr. Towler spent time treating patients who underwent these amputations. “I learned an awful lot from my patients,” he says. “Patients let you know what’s important to study. I would see something in the clinic and think, ‘I can take this to the lab and make a difference.’”

Dr. Dwight Towler

It’s people who have lost limbs or who have had heart valves replaced who keep him going. With obesity, diabetes, and kidney disease—risk factors for heart disease—on the rise, he feels that it is more important than ever to address these health issues. He adds, “As we live longer, the consequences of diabetes will be increasingly felt.” Dr. Towler also spent four years working in the pharmaceutical industry, where he contributed to the development of a drug for osteoporosis and other bone disorders. His study of bone development eventually led him to blood vessels. The same mechanism at work in the calcification of bones causes hardening of the arteries. This process sometimes “goes over to the dark side” as we age, says Dr. Towler. With the array of expertise and technology resources found at Sanford-Burnham, Dr. Towler feels very hopeful about the future of his research. “Orlando has a large clinical presence,” he points out. With abundant connections to other researchers, technology, and patients, he is ideally positioned to make an impact—to change the future for people with diabetes and heart disease. | PORTAL


Dr. Vincent Chen

Recreating a Person’s Unique Heart Disease in the Lab Consider This Scenario Brian loved to play football in the fall, basketball in the winter and baseball in the spring. The 18-year-old kept moving after practice, too, from pickup soccer games with friends to running 5K road races with his father. At least that was the case until one day last August, when Brian was halfway through a preseason football training session. He’d been working hard, but nothing out of the ordinary. Brian began to feel his heart fluttering. He felt dizzy and lightheaded. Suddenly, he collapsed. Brian was lucky that day. The paramedics responded quickly and resuscitated him. But he was later diagnosed with arrhythmogenic right ventricular dysplasia/cardiomyopathy (ARVD/C), an inherited heart condition. ARVD/C greatly increases Brian’s risk of sudden cardiac arrest and death. Brian survived, but his life is forever changed. He can no longer play sports and he’ll endure close medical supervision for the rest of his life—a life that could be unexpectedly cut short. Brian’s story is hypothetical, but real-life ARVD/C patients often don’t know they have a problem until their late teens or early 20s. Patients are usually diagnosed only at advanced stages of the disease or after sudden death—when it’s too late. This makes it especially difficult for researchers to study how ARVD/C evolves or to develop curative treatments. ARVD/C patients— often young athletes like Brian—frequently have to wear an electronic shock box to prevent sudden death, yet no treatment options are available to slow down the disease’s progression. Many ARVD/C patients eventually need a heart transplant. Disease in a Dish A new technique called “disease in a dish” might bring new hope to ARVD/C patients.



A research team led by Sanford-Burnham’s Huei-Sheng Vincent Chen, M.D., Ph.D., recently took simple skin samples from two ARVD/C patients. Then, using a stem cell-based technology that garnered a 2012 Nobel Prize, they converted the skin cells into heart cells. Now, living in a laboratory dish, these heart cells provide a unique model of each patient’s ARVD/C. The cells still carry the patients’ own genetic makeup and other unique characteristics. That makes for a very personalized way to study ARVD/C and test potential treatments. Speeding Up the Clock But using the “disease in a dish” model to study an adult disease can come with a major problem. Made straight from stem cells, Continued on next page


April 25, 6-9 p.m. Camp Bring It!

What’s your favorite memory of summer camp? Is it a great friend you made or a game you mastered? We’re giving you a chance to relive those fun, youthful memories with a grown-up purpose: raising money for stem cell research. You’re invited to our annual Bring It! event at the Del Mar Fairgrounds in Del Mar, Calif. Former San Diego mayor Jerry Sanders will co-chair with founding chairs Stath and Terry Karras. This year’s theme, Camp Bring It!, will challenge guests with a variety of camp-themed games. To purchase your table today, contact Karolyn Baker at 858.795.5239 or Or, register online at:

Remembering Esther Burnham Just before the new year, Sanford-Burnham said goodbye to a devoted friend and supporter, Esther Burnham. She gave generously to the Institute, as well as to a number of other organizations over many years. Sanford-Burnham benefactor Malin Burnham recalled his stepmother lovingly, saying, “Esther was a magnificent addition to our family. We were fortunate to have her in our family for almost 40 years.” In addition to medical research, Esther was a supporter of the arts, giving to organizations in San Diego and Los Angeles. She was also an avid sailor and excellent navigator who, with her late husband, sailed around the world. Sanford-Burnham scientists and staff will miss this vibrant and generous woman.

Continued from previous page

these newly made heart cells are mostly immature. That raises questions about whether or not they truly represent a disease that doesn’t appear until adulthood. “It’s tough to demonstrate that a diseasein-a-dish model is clinically relevant for an adult-onset disease. But we made a key finding here—we can recapitulate the defects in this disease only when we induce adult-like metabolism. This is an important breakthrough considering that ARVD/C symptoms usually don’t arise until young

adulthood. Yet the stem cells we’re working with are embryonic in nature,” said Chen. For that reason, Chen’s model is likely more relevant to human ARVD/C than other models. Chen and his team published their findings January 27 in the journal Nature. Dr. Chen was recently awarded a new grant from the California Institute for Regenerative Medicine to create more ARVD/C models. Together with collaborators at Johns Hopkins University, Chen also hopes to conduct preclinical studies aimed at finding new therapies for this deadly heart condition. | PORTAL



Marching Towards a Cure

Guests at the 2012 Sanford-Burnham gala sang, danced, and marched to the beat, led by our own “music man,” Conrad Prebys. The evening’s theme, Marching Towards a Cure, drew on elements of the classic Broadway musical The Music Man to make for an unforgettable evening. The event co-chairs (pictured below) were Conrad Prebys and Debbie Turner, Blair Blum and Jim Sexton, and Sheila and Jeffrey Lipinsky. Life Technologies was the lead sponsor.

Peter Preuss, Jr. and his wife, Erin, with Peggy and Peter Preuss



Malin and Roberta Burnham with longtime friends Gary and Jeanne Herberger

Events Leukemia and Lymphoma Society

Congressman Scott Peters

Dr. Louis J. DeGennaro, Erin Kozaki, Michael Copley, Dr. Vuori, Dr. Jackson, Malin Burnham, Dr. Ware, Dr. T. C. Chung, Kathlene Seymour.

Shea Benton, Scott Peters and Dr. Ze’ev Ronai

The executive senior management of the Leukemia and Lymphoma Society met with Drs. Kristiina Vuori, Michael Jackson and Carl Ware to discuss future innovative collaborative initiatives benefitting both organizations.

Sanford-Burnham was thrilled to host California Congressman Scott Peters, who was recently sworn in for his first term in Congress. Scientific Director Ze’ev Ronai, Ph.D. led him on a tour of our La Jolla campus.

Ferrari Party

More than 60 guests gathered in the home of Skeets and Sharon Dunn to learn about SanfordBurnham, hear from Dr. John Reed, and admire Skeets’ breathtaking collection of Ferraris.

New friends of Sanford-Burnham, Norman and Beth Saks

Trustee Brent Jacobs and wife, Joan | PORTAL


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Sanford-Burnham Medical Research Institute


Dr. Hudson Freeze and the Might Family

When their grandson, Bertrand, was found to have an extremely rare genetic disorder—so rare, in fact, he may be the only person in the world with it—Diane and Thomas Might reached out to Sanford-Burnham’s Dr. Hudson Freeze. Dr. Freeze is an expert on rare genetic diseases and a leading glycobiology researcher. Glycobiology is the scientific field that studies how cells coat proteins with sugar molecules—the process that is malfunctioning in Bertrand’s cells. The Mights chose to support the Freeze lab in order to help the team unravel Bertrand’s unique disease. They are hopeful, of course, that this research will help Bertrand. They are confident that they are advancing science to help numerous others, as well.



Spring 2013  

Join Sanford-Burnham Medical Research Institute, be a part of the quest to cure disease.

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