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UNC/Duke Team Publish on Using CRISPR Technique on Adult Mammals

Charles Gersbach, PhD & Aravind Asokan, PhD
Charles Gersbach, PhD & Aravind Asokan, PhD

A team of investigators supported in part by a $50,000 CTSA-supported Duke/UNC Collaborative Pilot Program has published results of their research into using CRISPR to treat an adult mouse model of Duchenne muscular dystrophy using a virus to deliver the gene-editing system. This marks the first time that the CRISPR technique has successfully treated a genetic disease inside a fully developed living mammal with a strategy that has the potential to be translated to human therapy.

The paper appeared on December 31, 2015 in Science. Duke's Charles Gersbach, PhD, and UNC's Aravind Asokan, PhD, who received the Duke/UNC Collaborative PIlot Program award in January 2015, are among the authors.

UNC-Duke Collaboration Probes the Depths of Gut Health

The key to physical and mental wellbeing could lie in your gut. The cells that line your digestive tract – all thirty feet of it – do more than just help your body digest food and get rid of waste. They stimulate your immunity, process toxins and pharmaceuticals, and even release hormones to ease anxiety and depression. But they aren’t all human.

Scott Magness, PhD & John Rawls, PhD
Scott Magness, PhD & John Rawls, PhD

The gut contains trillions of microbes, which are estimated to outnumber human cells by ten to one. Understanding how these diverse microbial communities – collectively known as the microbiome – influence human health is one of the next great frontiers for scientists, and a challenging one at that.

UNC stem cell expert Scott Magness, PhD, and Duke microbiome researcher John Rawls, PhD, are using a $50,000 grant to develop a new technology to study the co-dependent relationship between the human gut and its resident bacteria. The funding is part of an effort by the neighboring CTSAs to foster collaborations that accelerate the pace of biomedical research.

UNC and Duke Team Up to Fund Inter-Institutional Collaborations in Translational Research

Flag depicting UNC and DUKE

When people think about UNC and Duke, they often think about basketball rivalries, win-loss records, and March Madness. Collaboration is probably not the first thing that comes to mind. But outside athletics, these two schools are pooling their resources to create new teams to turn basic scientific discoveries into advances in patient care.

The North Carolina Translational and Clinical Sciences (NC TraCS) Institute and the Duke University Translational Medicine Institute (DTMI) are launching a new program to award $50,000 grants for translational research projects that involve a lead investigator from UNC and a lead investigator from Duke.

UNC and Duke Researchers Collaborate to Predict Drug Safety in Preemies

Children are not simply little adults. Not only do they not behave like adults, but their bodies are vastly different, with more bones, thinner skin, and narrower airways. And because of their faster metabolisms and smaller blood volumes, children often process drugs differently than grown-ups. These differences are particularly striking with premature infants, whose tiny physiques are even more susceptible to adverse reactions. Yet most of the drugs prescribed today for children and preemies have not been tested in those populations.

Christoph Hornik, MD, MPH & Daniel Gonzalez, PharmD, PhD
Christoph Hornik, MD, MPH & Daniel Gonzalez, PharmD, PhD

A research team from UNC and Duke has received a $50,000 grant to develop a new method for predicting drug safety in pediatric patients. The award is part of an effort by the neighboring CTSAs to promote collaborations that translate scientific discoveries into advances in patient care.

Daniel Gonzalez, PharmD, PhD, an assistant professor in the UNC Eshelman School of Pharmacy and Christoph Hornik, MD, MPH, an assistant professor in the Duke School of Medicine, will combine their expertise in pharmaceutical sciences and pediatrics to address

UNC and Duke CTSAs Award First Pilot Grants for Collaborative Translational Research Projects

UNC Duke awardees
Christoph Hornik, MD, MPH, Assistant Professor, Duke School of Medicine (left), Daniel Gonzalez, PharmD, PhD, Assistant Professor, UNC Eshelman School of Pharmacy (right)

The North Carolina Translational and Clinical Sciences (NC TraCS) Institute and the Duke University Translational Medicine Institute (DTMI) have awarded the first round of $50,000 translational research pilot grants to four teams with investigators from both UNC-Chapel Hill and Duke.

The grants are part of an effort to promote inter-institutional collaborations that can turn basic scientific discoveries into advances in patient care. Such collaborations can help accelerate the pace of research by granting more investigators expanded access to resources, expertise, and patient populations.

According to Jennifer Li, MD, co-principal investigator for Duke’s CTSA, the variety of scientific disciplines covered by the applications was stunning.

UNC and Duke Combine Forces to Repair Genetic Defects in Muscular Dystrophy

Charles Gersbach, PhD & Aravind Asokan, PhD
Charles Gersbach, PhD & Aravind Asokan, PhD

Anyone who has been sickened by the flu or a bout of food poisoning knows that viruses and bacteria can be powerful adversaries. But in recent decades, they have also become unlikely allies as scientists have turned bits and pieces of these infectious agents into tools for understanding and possibly even rewriting the human genome.

Researchers have gutted and replaced the insides of viruses with human genes, fashioning miniature couriers that can travel into cells to replace defects that underlie genetic diseases like hemophilia. They have also taken advantage of bacteria’s ability to recognize and chop up the DNA of invading pathogens, hijacking this defense system to edit the genome of any cell or species at will.

Tracking the Spread of HIV's Drug Resistance in NC

Ann Dennis, MD and Nwora Lance Okeke, MD, MPH / photo credit: Jon Gardiner
Ann Dennis, MD and Nwora Lance Okeke, MD, MPH photo: Jon Gardiner

In the 1990s, a person who became infected with the human immunodeficiency virus (HIV) could progress to full-blown AIDS within a few years' time. While there is still no vaccine or cure available for HIV, with early diagnosis and antiretroviral therapy (ART), infected individuals today can expect to live a long and relatively healthy life. And yet, an estimated 15 to 20 percent of people newly infected with HIV already carry a strain with mutations that allow the virus to resist some treatment options, according to Ann Dennis, MD, an Assistant Professor of Infectious Diseases at UNC.

Dennis has teamed up with Nwora Lance Okeke, MD, MPH, a Medical Instructor in Infectious Diseases at Duke, to track the spread of these resistant mutations across North Carolina over time.

The Power of Blood

NC TraCS researcher's unique expertise could one day thwart America's top killer

Alisa Wolberg
Alisa Wolberg

A conversation with Alisa Wolberg is not for the faint of heart. That's not to say that the trained biologist's demeanor is particularly fearsome: quite the contrary. She is enthusiastic and knowledgeable, generous with tidbits of scientific trivia and history whenever she alights on her favorite topic. The problem — some might find at least — is that Wolberg's favorite topic is blood. How it travels through hundreds of circuitous miles of arteries, veins, and capillaries to deliver oxygen and vital nutrients to every cell in the body. How a rare bleeding disorder passed down through generations of royalty helped to destroy Russia's Imperial Empire. How the opposite problem — dangerous blood clots known as venous thrombosis — strikes more than a million Americans each year.

Wolberg's laboratory studies the molecular, cellular, and biochemical mechanisms governing the formation of blood clots. She is intimately familiar with the coagulation cascade, the sequence of events that on paper look like a stone skipping across the surface on a pond, but in actuality represent the lineup of different protein factors activated on the path from a cut to a clot. In recent years, she has used funding from UNC's Clinical and Translational Science Awards (CTSA) to focus on one of these proteins, called factor XIII.

New UNC-Duke Collaboration Applies Cystic Fibrosis Therapies to Heart Disease

Given sufficient time, many forms of cardiovascular disease turn into heart failure, our nation’s number one killer. The disease affects six million Americans and costs the health care system a whopping $39 billion a year. Despite intense efforts to discover new heart medications, none of the current therapies have been able to halt its relentless progression.

Martina Gentzsch, PhD & Dawn Bowles, PhD
Martina Gentzsch, PhD & Dawn Bowles, PhD

As a result, researchers have begun looking to other diseases for inspiration. Cystic fibrosis, for instance, is experiencing a golden age of drug development. Several new drugs are in the pipeline to correct specific disease-causing defects in the cystic fibrosis transmembrane regulator (CFTR), a protein needed to keep airways hydrated. These advances are so noteworthy that President Obama even mentioned them in his recent State of the Union address, saying “in some patients with cystic fibrosis, this approach has reversed a disease once thought unstoppable.”

New Funding Opportunity for Collaboration - Duke/UNC CTSA Translational Pilot Grants - Request for Applications

This T1/T2 pilot program is designed to encourage and facilitate novel clinical and translational research that applies or accelerates discovery into testing in clinical or population settings. Cross-disciplinary basic research addressing the development of therapies, diagnostics or devices applicable to human disease and clinical research/trials are areas of interest for these awards. Projects must demonstrate high translational potential with a clear path to subsequent grant support, new company formation, licensing, not-for-profit partnering, or other channels.

Mentorship Training: The importance of being able to hit ‘pause’ on cringe-worthy conversations

"Did you plan this pregnancy?" the academic mentor demanded of his post-doctoral researcher. "What about our project timeline?" His mentee blinked in surprise. She'd just announced exciting news, but the reaction she received was not what she'd expected.

How would you respond to a similar comment from a mentor? How would you respond to your own mentees in this scenario? These were the questions Theater Delta performers posed to a group of about sixty academics from local universities in late January. The postdoctoral researchers and faculty came together to participate in a mentoring workshop organized by the North Carolina Translational and Clinical Science (NC TraCS) Institute and Duke University Office of Research Mentoring. The two-day workshop brought together faculty from different disciplines and schools to improve their understanding of an important influence on many academic careers: mentorship.

Mentoring, even informally done among academics, can make or break a researcher's career. Female researchers and those from underrepresented minorities are particularly vulnerable to diminished career advancement when they have poor mentorship options. Good mentorship is valuable to everyone, not just to those in the early stages of a career. According to Mark Dewhirst, director of Duke Office of Research Mentoring, it's important to remember that you can be both mentor and mentee at every stage throughout your career. When done right, these relationships benefit everyone involved throughout their careers.

Giving Blood a Boost

Ian J. Welsby, MD BS (left) Jay S. Ravel, MD (right) / photo credit: Jon Gardiner
Ian J. Welsby, MD BS (left) Jay S. Ravel, MD (right)
photo: Jon Gardiner

Duke/UNC Team use CTSA pilot funding to explore whether ‘rejuvenated’ blood can reduce transfusion frequency for people with sickle cell disease.

In healthy people, donut-shaped red cells in the bloodstream carry oxygen and deliver it to all of the body’s tissues. But in people with the inherited condition known as sickle cell disease, red cells stiffen and take on a sickled appearance. Those inflexible cells can stick to each other and to blood vessel walls, causing blockages that interfere with oxygen delivery and lead to sudden crises.

To improve blood flow and prevent dangerous complications, many sickle cell patients receive blood transfusions on a monthly basis. But, say Ian Welsby, a cardiac anesthesiologist and intensivist at Duke, and Jay Raval, a pathologist and transfusion medicine expert at UNC, the red blood cells in stored blood become exhausted over time in ways that might limit their lifespan. Now, with funding support from the Clinical and Translational Science Awards (CTSAs) at Duke and UNC, Welsby and Raval are teaming up to explore whether an FDA-approved method for rejuvenating the red blood cells in banked blood might help to extend the time between transfusions for patients with sickle cell disease.

Duke/UNC Team Testing New Strategies to Prevent Clotting in Patients with Pancreatic Cancer

Nigel Mackman, PhD and Rebekah White, MD
Nigel Mackman, PhD and Rebekah White, MD

CTSA collaborative funding award supports mouse-model research

People with cancer are at increased risk for developing blood clots as DNA, micro-particles and other cellular debris spills from tumor cells into the bloodstream. Many cancer patients also undergo surgery, which increases their risk of clotting even more. But when it comes to lowering the risk for blood clots in patients with cancer, today's doctors face a difficult dilemma.

"When you take cancer patients for surgery, they are at extremely high risk [for clots]," says Rebekah White, an associate professor of surgery at Duke. "But the blood thinners we have available increase the risk of bleeding."

Duke-UNC CTSA Pilot Grant Expands to Include Population Health Research

Flag depicting UNC and DUKE

Duke-UNC CTSA Collaborative Pilot Grants now looking for projects that fall anywhere on the spectrum of translational research, from laboratory bench to population health.

The Duke and UNC-Chapel Hill CTSA programs are once again joining forces to offer up to $50,000 for research projects that include investigators from both ends of Tobacco Road. But this year’s request for applications has expanded the scope of the projects.

Rather than focusing only on laboratory science and early clinical trials work, the door has been thrown open to include projects that cover the full spectrum of translational research, from nascent discoveries at the laboratory bench to investigations of how medical practices affect policy and population health. The deadline for applications is Oct. 2, 2015.

Duke-UNC Collaboration May Lead to Drug Discovery Boom

Robert Lefkowitz and Bryan Roth
Robert Lefkowitz (left) and Bryan Roth (right). Photo by Duke Photography.

Nobel prize winner from Duke connects with colleague at UNC to study cell receptors

As health conditions go, allergies, hypertension, and ulcers wouldn’t seem to have a lot in common. But many of the drugs used to treat these and other conditions work in a surprisingly similar way: by fitting together like lock and key with one of about 1,000 proteins found woven at the surface of our cells, known as G protein-coupled receptors (GPCR for short).

About 30 percent of all FDA-approved drugs in use today work by turning particular GPCRs on or off, making this class of receptors the most common of all drug targets.

Duke-UNC brings collaboration, too

When the Duke’s men’s basketball team travels to the Dean E. Smith Center on Saturday evening, it will be walking into a raucous, hostile arena created by decades of rivalry.

But off the court and outside the Dean Dome, University of North Carolina and Duke researchers are teaming up in science labs and have their eyes set on making key medical advancements.

They put the longstanding rivalry on the backburner. Well, most of the time.

“If Duke loses, I’ll be very happy — and I’ll probably give John a hard time about it,” admitted Scott Magness, a UNC-Chapel Hill researcher specializing in stem cells and bioengineered tissue scaffolds. His research partner, John Rawls, works at Duke.

Changing Course, Finding a Home

Jay Raval, MD
Jay Raval, MD
photo by Lane Deacon / UNC Health Care

From his position as a transfusion medicine physician, Jay Raval, MD, collaborates with providers from across UNC Medical Center to coordinate the best treatments for patients, while also giving him the chance to study ways to improve that care. His efforts have earned him this year’s Woods Junior Faculty Award.

As an undergraduate at UNC, Jay Raval, MD, knew he was interested in becoming a doctor. He just didn't have a clear answer for why. As a star student, he could have coasted into medical school, but he needed to experience medicine outside of the classroom first – something to help him explain the path he found himself on.

During his junior year at UNC, Raval worked as an EMT in the evenings and on the weekends. And in between classes, he was the assistant to the school nurse at a Chapel Hill elementary school.

"Being an EMT and working with a school nurse are probably on the opposite ends of the intensity spectrum," Raval said. "But both experiences taught me different things about medicine, how to think quickly in stressful situations, and how to build bonds with patients."

Can Gene-Editing Tools Cure Chronic Hepatitis B?

Researchers from Duke and UNC team up to find an answer, with funding from the two institutions' NIH Clinical and Translational Science Awards (CTSA).

For hundreds of millions of people around the world with chronic hepatitis B infection, anti-viral treatments do a good job of keeping the virus under wraps. Anti-viral treatments are essential in slowing damage to the liver, reducing the chance of liver cancer, and helping people live longer. But in the vast majority of cases, there is no end to the infection. If a patient stops medication for any reason, the hepatitis B virus (HBV) re-emerges. The cause of its resilience is circular bits of DNA, called covalently closed circular DNA, or cccDNA, that lurk in the liver cells of infected patients.

Bryan Cullen, PhD (Duke) and Lishan Su, PhD (UNC)
Bryan Cullen, PhD (Duke) and Lishan Su, PhD (UNC)

"Treatment blocks new viral replication," said Lishan Su, a professor of microbiology and immunology at the University of North Carolina, Chapel Hill. "It doesn't touch this cDNA." When treatment stops, new virus is produced from these cccDNA templates, he continued. As a result, "you need almost lifetime (or very long-term) treatment."

Tackling Human Brain Tumors by First Growing Them in a Dish

UNC and Duke researchers receive CTSA Consortium Collaborative Pilot Award to develop a new way to study cancer

Dr. Donald Lo (left) & Dr. Al Baldwin (right).

For doctors and patients, the fight against cancer can be a lot like an exceedingly tricky version of the classic arcade game of whack-a-mole. You might beat back a tumor or part of a tumor, only to have another one pop up. To make matters worse, the "mallet" or treatment that successfully whacks the first tumor cells doesn't always work on those arising later. You might need a new strategy or even an entirely different drug. It's a tough game to win.

The reason it is often unsuccessful to apply a singular approach to beating cancer is that cancer cells evolve and change over time as cells divide and tumors expand. That ever-changing, increasingly heterogeneous nature of cancer has been a particular problem in effectively treating glioblastoma multiforme, a common and aggressive form of brain cancer. Even when treated with cancer-fighting drugs, only one out of three patients with glioblastoma multiforme are alive two years after their diagnosis.

"There might be a drug that works against the founding mutation in a tumor, but now this branch diverged and might then show resistance," explained Albert Baldwin, Professor and Associate Director of Basic Research at University of North Carolina at Chapel Hill's Lineberger Comprehensive Cancer Center.

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