Dr. Bradley Yoder and Dr. Matthew Might are the director and co-director of C-PAM. Additional members of the leadership team include each of the leads of the C-PAM core sections. These directors provide interdisciplinary leadership across multiple key areas that are essential for the generation of precision models.
Dr. Yoder (Director) brings over 25 years of experience running a research lab focused on cilia biology and how defects in cilia-mediated sensory and signaling activity contribute to disease and developmental abnormalities. His group uses a wide range of model systems in their studies, predominantly C. elegans and mice, but have recently expanded into rats and zebrafish. Dr. Yoder has an extensive history of directing large projects (Director/PI of the P30 Hepatorenal Fibrocystic Disease Core Center (HRFDCC), Co-director Animal Models Core in the HRFDCC) along with administrative experience (PI of the T32 Training Program in Cell, Molecular, and Developmental Biology, Founding Director Graduate Biomedical Sciences Graduate Theme in Cell, Molecular, and Developmental Biology, Chair of the Department of Cell, Developmental, and Integrative Biology). He has analyzed the pathogenicity of multiple variants identified in human patients by targeting the alleles in mice as well as in C. elegans.
Dr. Might (Co-director) is the Director of the Hugh Kaul Precision Medicine Institute, Endowed Chair of Personalized Medicine, and Professor of Internal Medicine and Computer Science. His expertise is using precision medicine approaches such as artificial intelligence to predict therapeutic strategies for rare diseases, cancer, and common/chronic conditions. Dr. Might was a Strategist in the Executive Office of the President in The White House working primarily on President Obama’s Precision Medicine Initiative with both the NIH and VA. He was faculty in the Department of Biomedical Informatics at the Harvard Medical School with a focus on rare disease gene discovery, diagnosis, and personalized therapeutics. Dr. Might also has a personal story regarding his journey from computer science to medicine inspired by his son Bertrand. In 2012, Bertrand became the first patient in the world to be diagnosed with NGLY1 deficiency. Dr. Might’s social media and community network efforts led to the development of two therapeutics for NGLY1 deficiency. He is co-founder and Chief Scientific Officer of NGLY1.org, a non-profit dedicated to finding new ways to treat NGLY1 deficiency. He was a co-founder and Scientific Advisor to Pairnomix, a start-up that identifies potential patient-specific therapies for rare disorders – particularly genetic epilepsies. This combination of computer and computational science expertise, personal experiences in dealing with a rare disease, and public work has made Dr. Might renowned in rare disease research and advocacy.
Dr. Korf is the Pre/Co-Clinical Section (PCS) lead. He is a Professor of Genetics, Associate Dean for Genomic Medicine, and Chief Genomics Officer at the UAB School of Medicine; and was the previous chair of the Department of Genetics. Dr. Korf is the Co-Director of the UAB-HudsonAlpha Center for Genomic Medicine. He is a medical geneticist, pediatrician, and child neurologist, certified by the American Board of Medical Genetics (clinical genetics, clinical cytogenetics, clinical molecular genetics), American Board of Pediatrics, and American Board of Psychiatry and Neurology (child neurology). Dr. Korf was the past president of the Association of Professors of Human and Medical Genetics, the past president of the American College of Medical Genetics and Genomics, and is now the current president of the ACMG Foundation for Genetic and Genomic Medicine. He has served on the Boards of Scientific Counselors of the National Cancer Institute and the National Human Genome Research Institute at the NIH. He chairs the Medical Advisory Committee of the Children’s Tumor Foundation and serves on the CTF Board of Directors. He is also editor-in-chief of the American Journal of Human Genetics. He brings important clinical genetics expertise to the leadership team of the C-PAM.
Dr. Worthey is the Bioinformatics Section (BIS) lead. Dr. Worthey is an Associate Professor in Pediatrics and Director of the Center for Genomic Data Sciences in the Departments of Pediatrics and Pathology in the School of Medicine, the Director of the Bioinformatics Section in Pathology in the molecular diagnostics Division of Genomics Diagnostics and Bioinformatics, and an associate director for the Hugh Kaul Precision Medicine Institute. Dr. Worthey has spent the last 12 years developing and making use of tools for the identification and interpretation of molecular data to support definitive diagnosis and understanding of the genetic underpinnings of disease. She developed some of the earliest tools used for these purposes and is an internationally recognized expert in this area. These tools have been used to diagnose more than a thousand rare disease patients. In the leadership team for C-PAM, Dr. Worthey will provide expertise on computational methods and software solutions for sharing and analysis of C-PAM data.
Dr. Kesterson is the Disease Modeling Unit (DMU) lead. Dr. Kesterson is a Professor in the Department of Genetics, Director of the UAB Transgenic & Genetically Engineered Models Core (TGEM), Co-director of the In Vivo Bioassay and Model Development Resource in the P30 Hepatorenal Fibrocystic Disease Core Center. Dr. Kesterson provides expertise in generating targeted and CRISPR/Cas9 knockout, knock-in, and conditional mutant mice, rats, and zebrafish. He will guide engineering and screening strategies for all new animal models generated.
Dr. Wallis is the Resource and Services Section (RSS) lead. Dr. Wallis is an Associate Professor in the Department of Genetics. She has over 20 years of experience in using cell and animal models to evaluate gene function. She has directed validation, preclinical ADME-Tox, and efficacy studies and served as the primary investigator on several drug discovery projects that have generated novel compounds that are ready for human clinical trials. Currently, she runs a productive research program focused on Neurofibromatosis Type 1 (NF1), a RASopathy, that causes learning disabilities, macrocephaly, optic glioma, disfigurement, congenital abnormalities of the bone, scoliosis, and/or hypertension, and an increased risk of developing malignant tumors. Her lab has created numerous patient-specific cell line models to test genome-directed therapeutics including antisense oligos to mask cryptic splice sites, exon skipping, nonsense suppression therapeutics, and genome editing. Dr. Wallis will lead efforts on bioassay development using in vivo and cell-based models.
