Dr. Christina Grant

Children's National Health System, Washington DC, USA

Dr. Christina Grant is the Co-Director of the Lysosomal Storage Disease Program at Children’s National Medical Center where she is involved in patient care, clinical trials, and graduate medical education. Her focus is on both general genetics and patients with metabolic disorders, with a focus on lysosomal disorders and a special interest in gene therapy.

Dr. Grant completed her MD, PhD the University of Connecticut, with a degree in Cell Biology in 2012. Her graduate work focused on the interactions between cancer and blood vessels in cancer progression, and on how a specific molecule involved in prostate cancer was also necessary for abnormal blood vessel growth in other non-cancer diseases. She completed her internship and residency in Pediatrics at the Ann & Robert H. Lurie Children’s Hospital of Chicago (affiliated with Northwestern University) in 2015.

Dr. Grant developed a special interest in inborn errors of metabolism, especially lysosomal storage diseases, during her residency in Medical Genetics through the National Institutes of Health/ Johns Hopkins Combined program in Bethesda, MD. This was followed by a Fellowship in Medical Biochemical Genetics through the NIH, which she completed in 2018. Her research focus included lysosomal disorders and gene therapy.


DAY 3: September 13, 2019 | 11:45 AM - 12:00 PM

Patient Stories and How They Drive Systems Medicine Research

Christina Grant, MD, PhD, Children's National Medical Center, Washington DC, USA

Rare disease medicine faces the difficulty of small patient numbers for trials and for determining cause or treatment for disease. Knowledge of biological systems combined with bioinformatics can help in treatment and diagnosis of rare disease. Rare diseases also offer the biomedical science field a unique insight into cellular networks and mechanisms which is not as easily recapitulated in vitro or in silico due to the complexities of biologic systems, and can inform pathophysiology and therapy for more common conditions.

Examples of both biologic and informatics systems to understand rare disease include:

-repurposing a drug used in rare disease to treat far more common disorders based on biologic networks

-using a common medication to treat a rare disorder

-using bioinformatics to help diagnose rare genetic conditions

-tailoring therapy to the patient based on knowledge from both bioinformatics and biologic networks

As data analysis tools improve, it is expected that diagnosis and understanding of rare disease will also continue to advance.