Meet 2020 Uplifting Athletes Young Investigator Draft presented by CSL Behring research grant recipient Dr. Cheng Cheng


The Young Investigator Draft presented by CSL Behring is the result of Uplifting Athletes’ ongoing commitment to cultivate resources that accelerate scientific advancements for rare disease treatments and potential cures while facilitating the next generation of rare disease researchers.

To learn more about the 2020 Young Investigator Draft and to purchase tickets click here.

Researcher: Dr. Cheng Cheng

Young Investigator Institution: University of California, Irvine

Education: Dr. Cheng received her undergraduate degree from Knox College with a BA in Biology and Chemistry in 2013 and obtained her PhD in Developmental Biology from Washington University in 2018. Currently, Dr. Cheng works in the laboratory of Dr. Kimonis at UC-Irvine doing postdoctoral research.

Research: Dr. Cheng is conducting her PhD studies on the pathogenesis of Borjeson-Forssman-Leymann syndrome (BFLS), a rare syndromic form of X-linked intellectual disability. Using CRISPR/Cas9 technology, her research team has successfully generated and characterized the first rodent model of BFLS. This work advances understanding of the cellular and molecular underpinnings of BFLS and lays the foundation for potential treatments for neurodevelopmental disorders of cognition. In the Kimonis lab as a postdoctoral fellow, Dr. Cheng is exploring potential therapeutic strategies for VCP multisystem proteinopathy, a rare neuromuscular disorder. In particular, she is using small inhibitors to target the gain of function mutations of VCP protein in rodents and other cell models of the VCP disease. She is gaining experience in translational research in drug discovery by working with not only research scientists, but also clinicians, patients, pharmaceutical companies and patient advocacy groups. Her interactions with a wide variety of groups has taught Dr. Cheng about rare neuromuscular diseases from different perspectives, allowing her to effectively communicate science to a broad audience.

In Their Words: “Growing up in China, they have an interesting system. In high school you have to choose a path focused on either science or art classes. At the time I had to choose, I was pretty good at science stuff. So my high school was very science focused. When I came to the United States, I was inspired by my professors in their passion for research and the research they were doing. And that inspired me to look harder at research and I enjoy it very much.” – Dr. Cheng Cheng

Originally from the Citroen Province in China, Dr. Cheng came to the United States in 2009 to advance her love for science as a college student.

When she came to the U.S., Dr. Cheng wasn’t exactly sure what her path would be in science, but she was pretty sure she would find something that moved and inspired her.

You could say being a research scientist found Dr. Cheng, instead of the other way around.

“When I came to the U.S. I wasn’t sure exactly what in science I would do, but I felt strongly it was the right direction for me,” said Dr. Cheng, who has been married for three years and has a 2-year-old daughter and a 3-month old son. “I was so inspired by my professors and their passion for research and the research they were doing. That inspired me to look more closely at research.”

Focused on learning the techniques and details of being a research scientist, Dr. Cheng was drawn to neuroscience while working on her PhD. The chairman of the neuroscience at Washington University was studying rare intellectual neuroscience and Dr. Cheng was drawn to the work.

“When I think about a disease, there are two aspects. There is the science aspect and the humanity aspect,” said Dr. Cheng, who has traveled extensively and lists scaling Mount Kilimanjaro in Tanzania as one of her top accomplishments. “You learn something different from every patient. The science has a process, but you have to learn about the disease from a patient. That is very important to me.”

After more than a half decade in labs focused on honing her skills as a research scientist, it is meeting patients and understanding the value of translational science that continues to inspire Dr. Cheng.

The first time Dr. Cheng met an actual patient associated with the research she is working on, she was moved to tears. 

Because of her experience and commitment, last year Dr. Cheng was part of the inaugural Cure VCP Disease Patient and Caregiver Conference in St. Louis.

“The work we are doing is for patients with a very rare disease,” Dr. Cheng said. “We are doing more translational research and get to interact with the patient community. That is very important to me.”

Dr. Cheng was nominated for a 2020 Uplifting Athletes’ Young Investigator Draft research grant on behalf of Cure VCP Disease. Formed in 2018, Cure VCP Disease was formed to drive efforts to cure diseases related to mutations of the Valosin Containing Protein gene by providing global education and awareness of VCP diseases to doctors, researchers, patients, caregivers, investors and the general public.

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