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Gene therapy researchers find viral barcode to cross the blood-brain barrier

AAV therapy affecting pyramidal neurons in the hippocampus
This image shows AAV therapy affecting pyramidal neurons in the hippocampus. (Blake Albright, Asokan Lab)

UNC School of Medicine scientists led by Aravind Asokan, PhD, reveal how certain gene-carrying AAV vectors can penetrate the brain more efficiently to treat brain and spinal cord conditions, while reducing liver payload.

CHAPEL HILL, NC – Gene therapies promise to revolutionize the treatment of many diseases, including neurological diseases such as ALS. But the small viruses that deliver therapeutic genes can have adverse side effects at high doses. UNC School of Medicine researchers have now found a structure on these viruses that makes them better at crossing from the bloodstream into the brain – a key factor for administering gene therapies at lower doses for treating brain and spinal disorders.

"This structural 'footprint' we found seems to help these viruses get efficiently into the brain, which informs the design of potentially safer brain-targeted gene therapies," said study senior author Aravind Asokan, PhD, associate professor of genetics.

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Reprogrammed skin cells shrink brain tumors in mice

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Mouse and human skin cells can be reprogrammed to hunt down tumors and deliver anticancer therapies.
photo: Ella Maru Studio

Imagine cells that can move through your brain, hunting down cancer and destroying it before they themselves disappear without a trace. Scientists have just achieved that in mice, creating personalized tumor-homing cells from adult skin cells that can shrink brain tumors to 2% to 5% of their original size. Although the strategy has yet to be fully tested in people, the new method could one day give doctors a quick way to develop a custom treatment for aggressive cancers like glioblastoma, which kills most human patients in 12–15 months. It only took 4 days to create the tumor-homing cells for the mice.

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From Punnett Squares to Population Health

Kristin Young, PhD
Kristin Young, PhD

KL2 scholar Kristin Young fell in love in high school, not with a boy, but with a discipline. The first time she learned about Punnett squares — those beautifully simple 4x4 grids that enable geneticists to predict the inheritance of traits like eye color or diseases like cystic fibrosis — she was hooked. In college, she studied zoology, the only major at the time that included classes in genetics. Then she fell in love all over again when she took a class in anthropology, a field that transcended DNA and inheritance and took a holistic look at what makes us human.

Young went on to graduate school at the University of Kansas, where she combined her two loves to get a PhD in anthropological genetics. Today, she is a Research Assistant Professor of Epidemiology at UNC-Chapel Hill, where she studies the interaction between genes and environment and how that influences the human condition. Scientists have long debated the role of nature versus nurture in health and disease, and the more they learn, the clearer it becomes that most aspects of humanity cannot be solely attributed to one or the other.

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UNC thought leaders and practitioners convene to discuss Precision Medicine

Precision Medicine icon (UNC)

"Precision medicine gives us the chance to marry what's unique about America — our spirit of innovation, our courage to take risks, our collaborative instincts — with what's unique about Americans — every individual's distinctive genetic makeup, lifestyles, and health needs. In doing so, we can keep ourselves, our families, and our nation healthier for generations to come."

Those words, written by former President Obama and published in an op-ed in the Boston Globe last year, represent the lofty goal of precision medicine. Today, researchers at the University of North Carolina at Chapel Hill and other medical institutions are trying to operationalize that goal — to develop ways to target the right treatments to the right patient at the right time.

The UNC School of Medicine (SOM) and the NC TraCS Institute at UNC-Chapel Hill have facilitated a number of events to define the main areas of focus for these efforts.

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Michelle Maclay, Communications Director