Baylor College of Medicine’s Center for Precision Medicine Models has secured a five-year, $10 million grant from the National Institutes of Health. The funding will support ongoing research into rare genetic diseases through the development of specialized cell, fly, and mouse models that reflect patient-specific genetic variations.
The center’s approach enables researchers to investigate how specific genetic changes cause disease and to explore possible treatments. The initiative aims to improve understanding and care for individuals with rare conditions by integrating expertise in genetics, animal modeling, and bioinformatics at Baylor College of Medicine.
Dr. Jason Heaney, associate professor in the Department of Molecular and Human Genetics and lead principal investigator at the center, said: “The Department of Molecular and Human Genetics is uniquely equipped to bring together the diverse expertise needed to connect clinical human genetics, animal research and advanced bioinformatics tools. This integration allows us to drive personalized medicine forward using precision animal models and to turn those discoveries into better care for patients.”
The center operates an online portal where physicians, families, and advocacy groups can nominate genetic variants or rare diseases for further study or new treatment development. Submissions are reviewed by center experts for their potential impact and feasibility. Some cases are resolved during review; others proceed to further research or are referred elsewhere.
Since its establishment in 2020 with initial NIH funding, the center has received 156 nominations—63 were accepted for modeling—and has produced over 200 precision models. These efforts have led to the identification of new genes associated with rare diseases as well as new symptoms linked to known genes.
Dr. Lindsay Burrage, associate professor in the Department of Molecular and Human Genetics and a principal investigator at the center, stated: “The center’s success demonstrates the benefit of interdisciplinary collaboration to resolve variants of uncertain significance and develop therapies for rare disease.”
With continued funding, plans include expanding bioinformatic capabilities and extending research into complex diseases beyond rare disorders.
Additional co-principal investigators at the center are Drs. Aleksandar Milosavljevic and Shinya Yamamoto. Other affiliated faculty members include Drs. Hugo Bellen, Michael Wangler, Oguz Kanca, Denise Lanza, Audry Christinsen, Chih-Wei Logan Hsu, Pengfei Liu, Seema Lalani, Sandesh Nagamani, Matthew Roth, Uma Rammamurthy, and Jill Mokry.
This work is funded by National Institutes of Health Grant (U54 OD030165). More information about submitting nominations can be found on Baylor College of Medicine's website.
