The newly revamped Center, located in the Thurston Bowles Building, is buzzing with undergraduates and high school students looking to get a head start on their scientific careers.
Albert Einstein. Rosalind Franklin. Louis Pasteur. These renowned scientists have propelled humanity forwards through scientific innovation and out-of-the-box thinking. Statistically speaking, there are millions of “lost Einsteins” out there who have incredible intellect but lack the opportunities and support to pursue an academic career.
The Center for Molecular Medicine (CMM), formerly the UNC Gene Therapy Center, launched a new effort to give these potential Einsteins a foot in the door.
With support from the highly selective Amgen Scholars Program, the Carolina Summer Fellowship Program, and the WinSPIRE Summer Research Program, students are given the opportunity to conduct real-world research the Center’s affiliated laboratories.
The students come from colleges and universities across the United States, as well as high schools across North Carolina, to participate in biomolecular research right here at UNC. Most of the research revolves around adeno-associated virus (AAV) gene therapy—a type of therapy that uses a harmless virus to enter the body and make specific changes to someone’s genes.
Leading by Example
Samuel M. Young, Jr., PhD, the Director of the CMM, Roper Investigator, and professor within the Departments of Pediatrics, Pharmacology, and Microbiology and Immunology at the UNC School of Medicine, couldn’t be more excited to play a role in supporting the future scientists of tomorrow.
Young, himself a first-generation student, understands the challenges of university life.
“I was not prepared for college at all, and I faced many barriers,” said Young, who earned his undergraduate degree in Molecular Biology from Princeton University in 1996 and played defensive tackle on Princeton’s 1995 Ivy League Championship team.
One of those barriers came from within the Department itself. Young was told football players didn’t typically do research in molecular biology, which made him feel out of place. But then Arnie Levine, PhD, a renowned cancer researcher, offered him a chance. “I wouldn’t be where I am today without the opportunity and belief in my abilities without Arnie.”
Young has since inspired other labs in the Center to take in young scientists under their wing.
“How else can they know how to ask good questions, how to do rigorous research in a responsible manner, and to apply this to any problems that they encounter in their lives, if we don’t give them the opportunity?”
New Lessons Learned
Emma Tang is from Cary, North Carolina. She is a rising senior at UNC double majoring in chemistry and biology. As the daughter of an analytical chemist, her sights have always been set on studying drug delivery and drug optimization.
She reached out to many principal investigators to get some research experience under her belt. Some were full. Others never responded. She then “cold emailed” Chengwen Li, PhD, an professor in the Department of Pediatrics, and member of the Center for Molecular Medicine. When Tang asked to join the lab, Li replied with a “Yes, of course!”
The Li lab studies how AAV gene therapy can be improved, by finding ways to prevent the body’s immune response from attacking the virus. Tang’s specific research project focused on a signaling protein called glucagon-like peptide-1 (GLP-1), which has been shown to have a particular role in controlling immune responses and inflammation.
“We want to see if we can use [GLP-1] to try to prevent your immune system from destroying the virus that we’re trying to inject into your body to help you,” said Tang.
However, Tang was quite surprised by how lab work differs from lessons learned in a textbook.
“I’ve used almost none of the information I’ve learned in class in this lab,” said Tang. “But that’s not necessarily a bad thing. It just means that you have knowledge of both in-class information and real-life experience. But everyone’s there to help you and teach you things that you need to know, so you pick up things fast.”
Graduation is now on Tang’s horizon. As of right now, she is weighing on applying to graduate school where she can continue to learn and grow as an academic researcher or set forth on her father’s path in the pharmaceutical industry. Even if she gets rejected, she knows that, through sheer determination, she will find a way to pursue her passion.
Access to Top-of-the-Line Resources
Thomas McCown, PhD, is a professor of psychiatry and the Director of Basic Science at the CMM. When he isn’t studying the next treatment of neurological disorders, such as intractable temporal lobe epilepsy and Parkinson’s Disease, he is advising Amgen Scholars students like Ryan Mehlem.
Mehlem is a biochemistry major and undergraduate researcher at Clemson University in South Carolina. Hailing from Greenville, SC, Mehlem has long been interested in science, from making macgyvered inventions in his backyard to seeing gel electrophoresis in action in his middle school science class. It was only natural for him to pursue a degree in biochemistry.
At Clemson, Mehlem studies gene therapies that could help treat familial hypercholesterolemia, a genetic condition that causes high cholesterol. But there, he has limited access to AAVs, as they do not have the facilities to create the viruses for use in the lab.
In the McCown lab, he experienced how these innovative drug delivery tools are made and how to more effectively target the therapy so it only affects the desired cells. This summer, Mehlem found that changing some of the amino acids (protein building blocks) of a strain of AAV can give them different properties—a breakthrough finding that could help researchers to create more effective, low dose gene therapies.
“So just like how there are different strains of COVID or bacteria, there are different strains of AAVs, each with different properties,” said Mehlem. “The one I studied this summer is good at targeting neurons. The hope is we can find ways to make them be able to transduce cells better so that way we can use lower dosages with patients. If we can make something like twice as effective, then we can cut the cost in half and make gene therapy more accessible to everybody who needs it.”
Mehlem’s time in the McCown lab is thanks to the Amgen Scholars Program, a prestigious program which provides hundreds of undergraduate students to perform hands-on research experience at the world’s leading educational institutions. The program’s ultimate goal is to increase the number of students in advanced training and careers in the sciences.
With more experience under his belt, Mehlem is now applying to MD-PhD programs across the country, including UNC School of Medicine’s very own MD-PhD Program. One of his interests is to combine his passions for gene therapy and surgical delivery methods into one: by integrating targeted gene therapy into transplant surgery to make donor organs more compatible with recipients.
“A lot of the things that we are doing in the lab these days are incredible and have the potential to transform lives for the better,” said Mehlem. “The issue is just closing the gap. It’s really just a matter of finding the appropriate will and means to translate them into the clinic.”
Helping People Through Basic Research
Dulce Aguilar Cruz, a high school student from Graham High School in Graham, NC is heavily interested in helping people through the power of basic science.
“Since entering high school, I’ve always known that I wanted to help people in some way,” said Aguilar Cruz. “In the recent years, I’ve been thinking about going into radiation therapy. But, being in this lab exposed me to the different ways that you can help people.”
Through the WinSPIRE Summer Research Program, high school students take part in a paid six-week-long immersive summer mentorship program, where they are given hands-on experience in a research laboratory, engagement in college-prep workshops, and an extensive support network of mentors.
Aguilar Cruz trained under Tierra Bobo, PhD, an assistant professor of pediatrics and member of the Muenzer MPS Center at the UNC School of Medicine. Alongside Bobo, the duo helped develop novel gene therapy approaches to treat mucopolysaccharidosis III C, or MPS III C.
The inherited condition occurs when the body cannot break down a complex sugar called heparan sulfate, causing neurological complications, skeletal irregularities, enlarged organs, and more. Using AAV therapy, researchers are hoping to enhance the expression of a specific gene in individuals with MPS III C, to help a protein decompose the specific complex sugars that build up.
For Aguilar Cruz, the experience gained from the WINspire program has cemented her dreams of becoming a future Tar Heel.
“Going into this, I was really excited, because I actually want to go to UNC for college,” said Aguilar Cruz. “I’ve enjoyed being exposed to everything, at this lab and through WINspire. It’s been really cool just to see what this campus has to offer and I feel like it’s been more definitive for me that I want to go here.”
Paving an Academic Path for Biomedical Science
Nathaniel Conolly is a rising senior at the University of Iowa majoring in biology with a focus on neurobiology. Research interests him because he is always on the precipice of discovering something that has never been seen or done before.
As an undergraduate researcher in the lab of Samuel Young, Jr., PhD, Conolly was given the opportunity to do just that. This summer, he studied a variety of calcium ion channels, which play a crucial role in the communication between nerve cells, to see how their abundance is regulated in nervous system cells.
Conolly was a member of the Carolina Summer Fellowship (CSF) Program, which is sponsored by the UNC Department of Pharmacology and supported in part by a SURF award from the American Society for Pharmacology and Experimental Therapeutics (ASPET). Through the program, students planning a career in biomedical science conduct research under the direction of a research pharmacologist to gain knowledge and skills in scientific communication, networking, and foundational research techniques.
“I watched a lot of electrophysiology,” said Conolly. “And I got to learn how neurological data is collected and interpreted. As someone who is interested in neurobiology, these are skills that I want and need to have for the future.”
Conolly is actively applying to UNC Biological and Biomedical Sciences program (BBSP) to further his interests in neurobiology. His major takeaway from his summer experience: Choose a mentor that is passionate about teaching.
“I think that it’s important to find a lab, even if it’s not exactly where you’re interested in, that is passionate about teaching,” said Conolly. “So you know, you start your look more about the mentorship than the actual research, and then you can sort of continue the journey from there.”
For Young, that is what mentorship in the sciences is all about: providing confidence, support, and opportunity in a complex, yet highly rewarding field.
“We want to show we’re excited about our science,” said Young. “We’re excited about training the next generation and then giving opportunity. Because if we have 10 kids that come through, and two of them decide to go on to get their PhD, or they stay in science and become researchers, that’s a big win for us – and for humanity.”
Media contact: Kendall Rovinsky, Communications Specialist, UNC Health | UNC School of Medicine