Sanford-Burnham Science Blog
The first experimental drug to boost brain synapses lost in Alzheimer’s disease has been developed...
Sanford-Burnham will co-host the 9th annual World Stem Cell Summit December 4-6, 2013, in San Diego,...
On October 5, we opened our La Jolla campus to the San Diego community in honor of Stem Cell...
Sanford-Burnham researchers convince transplanted stem cell-derived neurons to direct cognitive...
Photomicrograph of nerve cell during an electrical recording (left), fluorescently labeled nerve cell (right)
The first experimental drug to boost brain synapses lost in Alzheimer’s disease has been developed by researchers at Sanford-Burnham. The drug, called NitroMemantine, combines two FDA-approved medicines to stop the destructive cascade of changes in the brain that destroys the connections between neurons, leading to memory loss and cognitive decline.
The winning team, from left to right: Brandon Heess, Kim Renna, Michele Bart, Christina McCabe, Nicole Lomitola, Ryan Hiller and Paul Jacobson in front.
On April 25, Sanford-Burnham hosted its fifth annual Bring It! event, the Institute’s spring fundraiser to benefit stem cell research. More than 200 guests attended the camp-themed affair which took place at the Del Mar Fairgrounds.
Sanford-Burnham will co-host the 9th annual World Stem Cell Summit December 4-6, 2013, in San Diego, together with The Scripps Research Institute, Genetics Policy Institute, Mayo Clinic, and California Institute for Regenerative Medicine.
Sanford-Burnham will co-host the 9th annual World Stem Cell Summit December 4-6, 2013, in San Diego, together with The Scripps Research Institute (TSRI), Genetics Policy Institute (GPI), Mayo Clinic, Kyoto University Institute for Integrated Cell-Material Sciences (iCeMS), and California Institute for Regenerative Medicine (CIRM). The large, multi-disciplinary conference features more than 170 experts, who will discuss the latest scientific discoveries, business models, translational issues, legal and regulatory solutions, and best practices.
Malene Hansen, Ph.D., assistant professor in our Del E. Webb Center for Neuroscience, Aging, and Stem Cell Research and native of Denmark, gave a faculty promotion seminar last week. It was her chance to show off her science and service to a committee that will evaluate her for promotion to associate professor. Check out her lab members in attendance. They’re showing their support with Danish flag scarves! Best of luck to Hansen and her team.
Scientific research isn’t always easy to explain—or to understand. Whichever side of the conversation you’re on, you might feel a communication gap. The California Institute for Regenerative Medicine (CIRM) , the state’s stem cell agency, recently set out to bridge that gap with a #SciencePitch Challenge. Their goal was to encourage stem cell researchers to develop their “elevator pitch” — the short overview of their work that they’d give if a fellow elevator passenger asked them what they do and they only had a short ride in which to explain it. In the process, the participants are also demonstrating the importance of stem cell research and generating excitement about their work.
Neurons from a normal mouse (left) are longer and fuller than neurons from a mouse lacking SNX27 (right).
Researchers discover that the extra chromosome inherited in Down syndrome impairs learning and memory because it leads to low levels of SNX27 protein in the brain.
What is it about the extra chromosome inherited in Down syndrome—chromosome 21—that alters brain and body development? Researchers have new evidence that points to a protein called sorting nexin 27, or SNX27. SNX27 production is inhibited by a molecule encoded on chromosome 21. The study, published March 24 in Nature Medicine, shows that SNX27 is reduced in human Down syndrome brains. The extra copy of chromosome 21 means a person with Down syndrome produces less SNX27 protein, which in turn disrupts brain function. What’s more, the researchers showed that restoring SNX27 in Down syndrome mice improves cognitive function and behavior.
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 Activity Center in Del Mar, Calif., on April 25. 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.
Neuron
In stroke and other neurological disorders, nitric oxide damages neurons and blocks the brain’s ability to self-repair
Nitric oxide, a gaseous molecule produced in the brain, can damage neurons. When the brain produces too much nitric oxide, it contributes to the severity and progression of stroke and neurodegenerative diseases such as Alzheimer’s. Researchers at Sanford-Burnham Medical Research Institute recently discovered that nitric oxide not only damages neurons, it also shuts down the brain’s repair mechanisms. Their study was published February 4 by the Proceedings of the National Academy of Sciences.
“In this study, we’ve uncovered new clues as to how natural chemical reactions in the brain can contribute to brain damage—loss of memory and cognitive function—in a number of diseases,” said Stuart A. Lipton, M.D., Ph.D., director of Sanford-Burnham’s Del E. Webb Center for Neuroscience, Aging, and Stem Cell Research and a clinical neurologist.
Lipton led the study, along with Sanford-Burnham’s Tomohiro Nakamura, Ph.D., who added that these new molecular clues are important because “we might be able to develop a new strategy for treating stroke and other disorders if we can find a way to reverse nitric oxide’s effect on a particular enzyme in nerve cells.”
In this study, researchers used an ARVD/C patient's skin cells to make induced pluripotent stem cells. Then they used those stem cells to generate ARVD/C patient-specific heart cells (shown here in green). These heart cells provide a valuable “disease in a dish” model that can be used to study ARVD/C and test new treatments.
Most patients with an inherited heart condition known as arrhythmogenic right ventricular dysplasia/cardiomyopathy (ARVD/C) don’t know they have a problem until they’re in their early 20s. The lack of symptoms at younger ages makes it very difficult for researchers to study how ARVD/C evolves or to develop treatments.
A new stem cell-based technology created by 2012 Nobel Prize winner Shinya Yamanaka, M.D., Ph.D., helps solve this problem. With this technology, researchers can generate heart muscle cells from a patient’s own skin cells. However, these newly made heart cells are mostly immature. That raises questions about whether or not they can be used to mimic a disease that occurs in adulthood.
In a paper published January 27 in Nature, researchers unveil the first maturation-based “disease in a dish” model for ARVD/C. The model was created using Yamanaka’s technology and a new method to mimic maturity by making the cells’ metabolism more like that in adult hearts. For that reason, this model is likely more relevant to human ARVD/C than other models and therefore better suited for studying the disease and testing new treatments.
Sanford-Burnham's Stem Cell Research Center provides resources and expertise to the entire scientific community. They are also building the world's largest collection of human induced pluripotent stem cells (iPSCs).
New collaboration combines Sanford-Burnham’s renowned scientific team and Intrexon’s proprietary discovery platforms to accelerate human induced pluripotent stem cell (iPSC) research
Today, we announced a new collaboration with Intrexon Corporation, a leading synthetic biology company, aimed at accelerating stem cell research. Under the agreement, Sanford-Burnham will gain access to sophisticated proprietary cellular selection and gene regulation technologies that are not currently on the market, including Intrexon’s Laser-Enabled Analysis and Processing (LEAP™) instrument and RheoSwitch Therapeutic System® (RTS®). As part of the agreement, Intrexon may obtain commercial and intellectual property rights resulting from technological advances made under the collaboration.
“I’m looking forward to merging and melding our expertise,” said Evan Y. Snyder, M.D., Ph.D., professor and director of Sanford-Burnham’s Stem Cell Research Center and Stem Cell and Regenerative Biology Program. “We’ll bring our iPSC and gene therapy expertise to the table. Likewise, our colleagues at Intrexon will share their knowledge of how best to use the technologies. We envision we’ll be meeting with them frequently and sharing insights to further advance the platforms for stem cell applications.”
Sanford-Burnham is currently building the world’s largest collection of human iPSCs generated from individual patients and healthy volunteers. The Stem Cell Research Center’s expertise and resources are available to all Sanford-Burnham scientists, as well as other researchers at nonprofit and for-profit research organizations around the world.
Stuart A. Lipton, M.D., Ph.D., director of Sanford-Burnham’s Del E. Webb Center for Neuroscience, Aging, and Stem Cell Research and a clinical neurologist
Originally published November 15, 2012
Researchers and patients look forward to the day when stem cells might be used to replace dying brain cells in Alzheimer’s disease and other neurodegenerative conditions. Scientists are currently able to make neurons and other brain cells from stem cells, but getting these neurons to properly function when transplanted to the host has proven to be more difficult. Now, researchers at Sanford-Burnham Medical Research Institute have found a way to stimulate stem cell-derived neurons to direct cognitive function after transplantation to an existing neural network. The study was published November 7 in the Journal of Neuroscience.
A consortium of researchers around the U.S. used transplanted neural stem cells (shown here) to treat a mouse model of ALS.
In 11 independent studies, a consortium of ALS researchers shows that transplanting neural stem cells into the spinal cord of an ALS mouse model slows disease onset and progression, improves motor function, and significantly prolongs survival.
Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, is untreatable and fatal. Nerve cells in the spinal cord die, eventually taking away a person’s ability to move or even breathe. A consortium of ALS researchers at multiple institutions, including Sanford-Burnham Medical Research Institute, Brigham and Women’s Hospital, and the University of Massachusetts Medical School, tested transplanted neural stem cells as a treatment for the disease. In 11 independent studies, they found that transplanting neural stem cells into the spinal cord of a mouse model of ALS slows disease onset and progression. This treatment also improves host motor function and significantly prolongs survival.
Surprisingly, the transplanted neural stem cells did not benefit ALS mice by replacing deteriorating nerve cells. Instead, neural stem cells help by producing factors that preserve the health and function of the host’s remaining nerve cells. They also reduce inflammation and suppress the number of disease-causing cells in the host’s spinal cord. These findings, published December 19 in Science Translational Medicine, demonstrate the potential neural stem cells hold for treating ALS and other nervous system disorders.
“While not a cure for human ALS, we believe that the careful transplantation of neural stem cells, particularly into areas that can best sustain life—respiratory control centers, for example—may be ready for clinical trials,” Evan Y. Snyder, M.D., Ph.D., director of Sanford-Burnham’s Stem Cell and Regenerative Biology Program and senior author of the study.
The latest episode of Developments to Watch, our collaborative video series produced by Medscape, is now available online: Disease in a Dish: The Ultimate Personalized Medicine.
In the video, Sanford-Burnham CEO John Reed, M.D., Ph.D., talks to Michael Jackson, Ph.D., vice president of drug discovery and development, about the Institute’s work on creating personalized “disease in a dish” models using stem cells derived from patients. They also talk about drug repurposing—finding new applications for existing therapeutic drugs in order to get treatments to patients faster.
Here’s an excerpt:
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.
The International Space Station, as seen from the departing Space Shuttle Discovery in 2009 (Image courtesy of NASA)
Space Florida to send two experiments from Sanford-Burnham Medical Research Institute to the International Space Station
We’re excited to announce today that two of our research teams have won Space Florida’s International Space Station (ISS) Research Competition. Eight teams were selected from a pool of international applicants to send experiments to space in late 2013. The competition was initiated by Space Florida, the state’s spaceport and aerospace authority, and NanoRacks, LLC. Sanford-Burnham’s research will fly as payloads to the ISS aboard a SpaceX Falcon 9 launch vehicle and research will be conducted on board the U.S. National Lab at the ISS.
Here’s what the two teams are hoping to accomplish:
