We are thrilled to feature Dr. Miao Cui, whose scientific journey bridges continents and disciplines—from evolutionary biology in China to cutting-edge heart regeneration research in the U.S.
Dr. Cui leads a lab focused on unlocking the regenerative potential of the heart, especially by studying how neonatal mice are able to recover from heart attacks—an ability lost in adulthood. Her team uses AAV-based gene therapy to understand and activate molecular pathways that promote tissue repair. One of her lab’s major discoveries includes identifying NRF1 as a key gene in neonatal heart regeneration, which regulates antioxidant defenses and protein homeostasis.
By combining tools like lineage tracing, CRISPR-Cas9, and spatial transcriptomics, Dr. Cui is uncovering how specific genes and cell states can be leveraged to heal the adult heart. Her current projects include high-throughput in vivo screening and mapping transcriptomic shifts across heart development, aging, and disease. Her ultimate goal? To translate these findings into therapies that help the human heart regenerate itself—no transplant required.
Outside the lab, Dr. Cui brings a creative eye to her work, inspired by a lifelong love of nature, math, and art. She even mused that if she weren’t a scientist, she’d be a painter or graphic designer—a nod to the beauty she sees in biology.
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We’d love to start with where you’re from! Please tell us about your background, and what lead you to a career in science?
Dr. Miao Cui: I grew up in China and moved quite a bit growing up. My family relocated between cities frequently, which gave me a broad exposure to different places and people. I did my undergraduate studies at Nanjing University, where I majored in zoology and evolutionary biology. Afterward, I pursued a master’s degree in evolutionary biology, also in China. In 2010, I came to the U.S. to begin my PhD in developmental biology at the California Institute of Technology. There, I studied gene regulatory networks in sea urchin embryonic development. Interestingly, that experience still influences how I think about gene regulation today.
That’s a big shift. What made you transition from sea urchins to heart research?
Dr. Miao Cui: Studying sea urchins was an excellent entry point into developmental biology. The system is clean, and you can learn a lot about fundamental mechanisms. But I increasingly felt the work was too far removed from human disease. What made the shift urgent and personal was the unexpected passing of my PhD advisor due to a heart attack during my final year at Caltech.
“The loss of my PhD advisor to a heart attack made me realize that I wanted to work on something that could have a more immediate impact on human health. That’s how I pivoted from sea urchins to the mammalian heart.”
That loss deeply impacted me and became a pivotal motivator in choosing a research path focused on cardiovascular biology. When I joined UT Southwestern for my postdoc, I started working on mechanisms that promote heart regeneration.
When did you know you wanted to become a scientist?
Dr. Miao Cui: It wasn’t a childhood dream—I wasn’t the kind of kid who declared, “I want to be a scientist!” I’ve always been curious, though. I loved nature, was drawn to math and logic puzzles, and I had a deep appreciation for the arts—especially painting.
“Science became the perfect blend of logic and creativity.”
Growing up in China, the college entrance exam system is incredibly competitive, and it shaped many decisions. I realized pursuing art professionally wouldn’t be practical. With encouragement from my parents and relatives—especially my mom, a math teacher—I chose biology. But my passion for research truly solidified during my graduate school years. It was a gradual process of discovery and affirmation.
What does your lab study, and how would you explain it to someone outside of science?
Dr. Miao Cui: At a high level, we study why newborn mammals can regenerate heart tissue and how we might reawaken that ability in adults. Specifically, our lab focuses on the regenerative potential of the neonatal heart.
“Nature has already solved the problem—we’re just trying to relearn the code.”
In mice, for instance, newborns can recover from severe heart injuries—like a heart attack—within a few weeks. That ability is lost as they age. We use genetic tools, especially AAV vectors, to explore the molecular and cellular mechanisms behind this phenomenon. Ultimately, we want to translate these discoveries into strategies that help adult hearts heal after injury.
Can you walk us through what happens during a heart injury experiment in your lab?
Dr. Miao Cui: We induce a myocardial infarction—essentially a simulated heart attack—by ligating the left anterior descending artery in neonatal mice. What’s amazing is that these mice can fully regenerate the damaged tissue within three weeks. Not only does the structure return to normal, but so does function—measured by metrics like ejection fraction. Adult mice, on the other hand, don’t recover in this way. They form permanent scar tissue, which leads to heart failure over time. Our mission is to understand why and figure out how to restore regenerative capacity.
Have you seen evidence of similar regeneration in human infants?
Dr. Miao Cui: Yes. There are rare but compelling clinical reports of human infants recovering from major heart injuries caused by congenital defects. These cases suggest that some regenerative potential exists in human newborns as well.
“We believe the key to healing adult hearts might be locked in the first few days of life.”
We’ve collaborated on studies analyzing single-cell RNA sequencing data from human neonatal hearts and found cardiomyocyte populations with similar molecular signatures to what we observe in mice. But these populations fade quickly with age, which parallels the loss of regenerative capacity.
What tools do you use to study this regenerative capacity?
Dr. Miao Cui: Our toolbox includes lineage tracing, spatial transcriptomics, and CRISPR-based barcoding. For instance, we use a system called the CARLIN mouse model, developed here at Boston Children’s Hospital, which allows us to label individual heart cells with a unique genetic barcode. That way, we can track which cells actually contribute to regeneration. Combined with transcriptomic data, we can see both the lineage and the gene expression profile of regenerating cells. This gives us a powerful view of how regeneration unfolds.
Can you talk about your work with the gene NRF1?
Dr. Miao Cui: NRF1 is a transcription factor that became a major focus for us because it’s highly expressed in the regenerative cardiomyocytes of neonatal mice. When we knocked out NRF1 in these cells, they lost the ability to regenerate. When we overexpressed it in adult hearts using AAV gene delivery, we saw improved recovery.
“NRF1 is like a personal trainer for stressed-out heart cells—getting them fit enough to divide and repair.”
Mechanistically, NRF1 regulates genes involved in oxidative stress response and protein homeostasis. It essentially helps cells stay healthy under stress, which is critical for them to proliferate and repair tissue.
What are the biggest hurdles to translating these findings to adult humans?
Dr. Miao Cui: Scalability and complexity. We’re generating large datasets from single-cell genomics that identify candidate genes but testing them one by one is inefficient.
“To fix adult hearts, we have to think big—and scale even bigger.”
That’s why we’re now building a high-throughput screening platform using barcoded AAV vectors. Each vector delivers a different gene to the heart, and we track which ones produce a beneficial effect. It’s a faster, more scalable way to move from discovery to therapy. We’re also studying how the heart’s transcriptome changes across the lifespan—from neonatal to aged—and asking whether we can reprogram aged or diseased hearts toward a more regenerative state.
Outside the lab, what are your interests?
Dr. Miao Cui: I still love painting. If I hadn’t become a scientist, I think I would’ve pursued visual arts or maybe even architecture. I find that engaging with art helps me think creatively in science too.
“Science and art are closer than people think. Both are about observing, interpreting, and imagining what’s possible.”
What scientific breakthroughs in the past decade have most inspired you?
Dr. Miao Cui: Single-cell genomics, hands down. It has completely transformed how we understand cellular diversity and gene regulation. I’m also excited by the potential of in vivo genomic screens. When combined, these technologies let us not only see what cells are doing but test how they respond to precise perturbations.
“Single-cell genomics is like giving biology a microscope for the soul.”
Where can people follow your work?
Dr. Miao Cui: We have a lab website where we share our publications and updates on ongoing projects. I used to be more active on Twitter but have shifted my attention elsewhere lately.