Children’s Health

Muscling up with MyoD

by Faculty Contributor on January 13, 2012 at 8:49 am | 1 comment

Lorenzo Puri, M.D., Ph.D.

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 rare approach for a rare disease

by Kristina Meek on November 23, 2011 at 12:29 pm | 2 Comments

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Mayor Hall of Bakersfield cuts the ribbon to open Salon Gianna. Beside him is Gianna, with Dr. Hudson Freeze and her mother, Natalie. On the right are Rocket’s parents, Mia and Taylor Williams, and Gianna’s father, David.

People who care about curing disease can be very creative in finding ways to raise money for research. Whether someone raises money and awareness by walking 60 miles, growing a mustache, or spending $50,000 on a pair of novelty sneakers, people’s passions fuel research funding.

The family that owns Salon Gianna, a beauty salon in Bakersfield, California, is on a mission to find a cure for Congenital Disorders of Glycosylation (CDG). All of their proceeds are earmarked for The Rocket Fund at Sanford-Burnham, which is overseen by Dr. Hudson Freeze. CDG is actually a group of more than 30 rare diseases caused by inherited defects in glycosylation, the process cells use to coat proteins with sugars. Young patients have a broad spectrum of clinical problems often including developmental delay, serious intestinal and liver complications, clotting defects, eye, skin, and other defects. Dr. Freeze’s lab seeks to treat and cure these diseases, often working closely with the families of affected children.

Sisters in science

by Heather Buschman, Ph.D. on November 4, 2011 at 9:41 am | 0 Comments

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Dr. David Pearce, Vice President of Research at Sanford Health and Director of Sanford Children’s Health Research Center in Sioux Falls

In late 2007, the Sanford Children’s Health Research Center was established at Sanford-Burnham’s La Jolla campus with a $20 million gift from South Dakota philanthropist T. Denny Sanford through Sanford Health. The gift was the foundation for a long-term partnership between Sanford Health, a large healthcare system based in South Dakota, and Sanford-Burnham. In addition to the center in La Jolla, in 2009 Sanford Health created a sister Children’s Health Research Center in Sioux Falls.

On October 27-28, researchers from both research centers gathered at Sanford-Burnham’s La Jolla campus to share new research directions and stimulate further collaboration at the fourth annual Sanford Children’s Health Research Center Scientific Symposium. Attendees heard overviews from the leaders of both Sanford-Burnham and Sanford Health and learned about Sanford Health’s new BioBank, a repository for patient samples that will help drive personalized medicine and provide fodder for population genomics studies. More than a dozen scientists presented their ongoing studies of embryonic development, type 1 diabetes, brain tumors, lung injury in newborns, and rare inherited conditions such as Batten disease. Hot topics also included stem cells and RNA biology.

An easier (and safer) route to stem cell therapies

by Bruce Lieberman on October 31, 2011 at 9:32 am | 0 Comments

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Chao-Shun Yang, graduate student in Dr. Tariq Rana's lab and first author of the study

Many research labs around the world are focused on finding the most effective ways to reprogram an adult cell (a skin cell, for example) into induced pluripotent stem cells (iPSCs)—that is, cells that have the ability to develop into other tissues in the body. These cells not only offer researchers powerful tools to study a particular patient’s individual disease, but they have the potential to therapeutically replace diseased or damaged tissue in the patient from whom the cells originated.

Most experiments to reprogram adult cells employ viruses as vehicles to carry four particular genes—called reprogramming factors—into the nucleus of a cell. But genetic engineering carries its own risks, including the chance that these cells will continue replicating, eventually forming a tumor. What’s more, scientists are not exactly sure what the reprogramming factors do, on the molecular level, to promote the generation of iPSCs.

Could there be a safer and more predictable way to alter the expression of genes in cells, thereby reprogramming their DNA so they revert to their earlier, more malleable state?

Save the Date: Third Annual Rare Disease Symposium

by Heather Buschman, Ph.D. on August 12, 2011 at 4:18 pm | 0 Comments

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Gianna Dragatto, a young patient with a rare disease called Congenital Disorders of Glycosylation (CDG), hopes to see you at Sanford-Burnham's 3rd Annual Rare Disease Symposium on February 24, 2012.

Dr. Eric Green, keynote speaker (photo by Maggie Bartlett)

What: 3^rd Annual Sanford-Burnham Rare Disease Symposium
Where: Sanford-Burnham Medical Research Institute, La Jolla, Calif.
When: February 24, 2012
Keynote speaker: Dr. Eric Green, director of the National Human Genome Research Institute (NHGRI) at the NIH

Sanford-Burnham’s successful series of Rare Disease symposia is based on the concept that treatment of rare diseases requires participation and exchange among all stakeholders—scientists, physicians, affected patients and their families, support groups, granting agencies, industry, and philanthropists.

This year’s event, organized by Dr. Hudson Freeze, will focus on Glycosylation-Based Disorders: Discovery, Patients, and Progress Toward Treatments.

Save the date now. Program and registration information will be available soon. In the meantime, video and media coverage of last year’s event are available here.

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Science…under the Tuscan sun

by Faculty Contributor on July 21, 2011 at 2:24 pm | 0 Comments

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Harrison Ford plays a glycobiologist in the movie Extraordinary Measures.

In the movie Extraordinary Measures, Harrison Ford plays a glycobiologist.

If brides and grooms can have destination weddings, then scientists should be able to have destination research conferences. These types of conferences are increasingly popular as opportunities for scientists to experience fun locales while also interacting and exchanging ideas with a relatively intimate group of expert colleagues. The Gordon Research Conferences have been trendsetters with this format since the 1930s, sponsoring scientific meetings on a variety of topics at sites within the U.S. Starting in 1990, Gordon Conferences have been held in more exotic foreign locations, including Italy, Switzerland, Japan, England, Hong Kong… and even Texas.

Earlier this summer, Dr. Hudson Freeze, program director in Sanford-Burnham’s Sanford Children’s Health Research Center, chaired the Gordon Conference on Glycobiology in Lucca, Italy. 170 glycobiologists from around the world gathered to hear about exciting new developments in the science of carbohydrates (sugar molecules) and the complex molecules like proteins and lipids whose properties are influenced by incorporation of carbohydrates. Once a rather understudied area of biology, glycobiology has been transformed by the realization that carbohydrates mediate many of the key molecular interactions that govern cellular function. Meeting topics included the effects of sugar modifications during development, the role of carbohydrates in normal adult physiology and the involvement of carbohydrates in tissue engineering and repair, including their importance in stem cell biology.

My moment with Corinna

by Heather Buschman, Ph.D. on May 27, 2011 at 6:39 am | 0 Comments

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This video of three year-old Corinna is remarkable because it would seem so unremarkable if you didn’t know Corinna’s story—she looks just like any other kid. I had the pleasure of meeting this very special attendee at Sanford-Burnham’s 2^nd Annual Rare Disease Day Symposium last February. Corinna was born with hypophosphatasia (HPP), a rare inherited disease that affects bone development, leaving her fragile and unable to walk. Lauren, Corinna’s mother, had brought her along to the symposium and was excited to meet the scientists studying HPP and hear about the latest research.

The family traveled from Philadelphia to be there that day – no small task, considering Corinna’s special needs and lack of mobility. I first talked to Lauren on the phone, helping her with directions from her hotel to the Sanford-Burnham campus. Then I waited for them outside and helped her get the stroller out of the taxi. I set it up while Lauren got Corinna out of the car. Corinna was blond, adorable, friendly—and just about the same size as my own daughter. My heart went out to them. I know how trying it can be to travel with a toddler, even under the best of circumstances.

A medical revolution

by Josh Baxt on May 18, 2011 at 8:04 am | 1 comment

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Nanoparticles, like this micelle, may be the future of medicine. (Image by Peter Allen, UC Santa Barbara College of Engineering)

A syndicated article that recently appeared in the Orlando Sentinel, the Los Angeles Times and other outlets described several revolutionary technologies that will change medicine in the coming decade.

In particular, the piece highlighted how new genomic technologies can personalize treatment to individual patients; how robotic surgery will help surgeons perform complex procedures on people thousands of miles away; and how new classes of diagnostic tests will allow physicians to discover diseases earlier, when they are most treatable.

The article included insights from Dr. Ranjan Perera, associate professor at Sanford-Burnham’s Lake Nona campus, and Dr. Jamey Marth, who directs the U.C. Santa Barbara–Sanford-Burnham Center for Nanomedicine. Dr. Marth is particularly excited about nanomedicine’s potential to enhance both diagnosis and treatment:

“Today’s scientists work at the molecular and atomic level with nanoparticles, to harness these biomachines that detect and bind to diseased cells. The nanoparticle then fuses with that sick cell and delivers its cargo — drugs or imaging agents.”

Read ‘Revolution is at hand’ for breakthroughs in medicine.

Where will new medicines come from?

by Heather Buschman, Ph.D. on May 16, 2011 at 5:00 am | 1 comment

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http://www.rejuveskinclinics.com/before_after_photos.html

National Institutes of Health (NIH) in Bethesda, Maryland

In December 2010, the National Institutes of Health (NIH) announceda plan to create a new center, known as the National Center for Advancing Translational Sciences (NCATS), to speed development and testing of novel diagnostics and therapeutics aimed at a wide range of human diseases.

In a recent opinion piece published in Genetic Engineering & Biotechnology News (GEN), Dr. Henry I. Miller, policy fellow at Stanford University’s Hoover Institution, argues against NCATS, claiming that drug development is better left in the hands of private pharmaceutical and biotechnology companies.

In a rebuttal published online May 13, Sanford-Burnham CEO Dr. John Reed picks apart Dr. Miller’s argument, explaining why the public sector must participate in the search for new medicines and how NCATS will further catalyze these efforts. According to Dr. Reed, private companies are increasingly reluctant to fund the crucial early stages of pre-clinical development – the research necessary to “translate” promising discoveries made in laboratories into potential therapeutics ready for testing in human clinical trials. He writes:

“This situation leaves us with the aptly named “Valley of Death” – the large research and funding gap that sets federally-funded basic researchers (those of us in non-profit research institutions, academia, hospitals and federal laboratories) on one side and the pharmaceutical industry on the other. Few companies are able to reach far enough backward to bridge that gap – the costs and risks are just too high for organizations that are responsible for delivering financial results to their investors and shareholders. Enter the NIH’s newly proposed center for translational sciences – just the shot in the arm basic research needs to reach forward across that valley.”

For more, read “NCATS Could Mitigate Pharma Valley of Death“, Genetic Engineering & Biotechnology News, online May 13, in print May 15, 2011.

One cell’s junk is another’s treasure

by Heather Buschman, Ph.D. on May 10, 2011 at 10:02 am | 0 Comments

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Melanoma cells, with nuclei in blue and SPRY4-IT1 in green. (Image courtesy of the Perera lab)

Melanoma cells, with nuclei in blue and the lncRNA SPRY4-IT1 in green. (Image courtesy of the Perera lab)

Scientists used to think RNA was mostly just a messenger molecule that carries protein-making instructions from a cell’s nucleus to the cytoplasm. But scientists now estimate that 97 percent of human RNA doesn’t actually code for proteins at all. A flurry of research in the past decade has revealed that some types of non-coding RNAs switch genes on and off and influence protein function.

The best studied non-coding RNAs are the microRNAs, but Dr. Ranjan Perera and his collaborators are discovering that levels of a relatively understudied group of RNAs – long, non-coding RNA (lncRNA) – are altered in human melanoma. Their study, published online May 10 by the journal Cancer Research, shows that one lncRNA called SPRY4-IT1is elevated in melanoma cells, where it promotes cellular survival and invasion.

“Non-coding RNA used to be considered cellular junk. But we and others have been asking the question – if it doesn’t code for proteins, what does it do in the cell?” said Dr. Perera, associate professor at Sanford-Burnham’s Lake Nona campus in Orlando, Fla. “We’re especially interested in determining what roles microRNAs and lncRNAs play in the genesis and development of human melanomas.”

Melanoma is one of the rarest forms of skin cancer, but it is also the most deadly.

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 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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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

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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.

Quick peek at Sanford-Burnham

by Josh Baxt on March 23, 2011 at 7:19 am | 0 Comments

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Sanford-Burnham's La Jolla campus. Photo by Nadia Borowski Scott

A video by high school student Daniel Osterman, son of Sanford-Burnham investigator Dr. Andrei Osterman, takes a quick look at the basic biomedical research being conducted at the Institute. In particular, the piece focuses on Dr. Hudson Freeze’s research. Dr. Freeze recently organized Sanford-Burnham’s 2^nd Annual Rare Disease Symposium, and studies a group of rare conditions called Congenital Disorders of Glycosolation (CDG), in which sugars fail to attach properly to proteins.

Rare disease symposium has uplifting moments

by Josh Baxt on March 4, 2011 at 11:04 am | 0 Comments

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Kate Fischer with daughter Morgan, who is being treated for HPP.

There was a moment at our recent Rare Disease Symposium when Dr. Michael Whyte, a pediatrician from Shriner’s Hospitals in St. Louis, presented video of a patient who is participating in a clinical trial. The patient, Amy, suffers from hypophosphatasia (HPP), a genetic bone disease similar to rickets. The trial is for an enzyme replacement therapy developed collaboratively by Dr. Whyte, Dr. José Luis Millán and Enobia Pharma to treat HPP. Before treatment, Amy’s bones were so soft she had to be flown to the trial in an insulated box. She was weeks away from dying. In the video, she runs, jumps and kicks a ball. Hard not to be moved.

Enobia’s HPP drug is in Phase II clinical trials and looks quite promising. However, rare diseases present a difficult problem. While relatively few people suffer from any single rare disease, there are thousands of these conditions. Large pharmaceutical and biotech companies have a difficult time addressing them because they have not figured out how to make back their investments. But the issues go even deeper. How do you conduct a robust clinical trial on a new treatment when only a handful of people need to be treated? And how do you balance the regulatory environment to ensure that new, safe treatments can reach patients? In fact, how do you even diagnose a rare disease when so few physicians have any experience with it?

Top Stories - Children's Health

Muscling up with MyoD

A rare approach for a rare disease

Sisters in science

An easier (and safer) route to stem cell therapies

Save the Date: Third Annual Rare Disease Symposium

Science…under the Tuscan sun

My moment with Corinna

A medical revolution

Where will new medicines come from?

One cell’s junk is another’s treasure

A few minutes with Mark Mercola

Leaders among peers

Personalized Medicine 101

Quick peek at Sanford-Burnham

Rare disease symposium has uplifting moments

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