Speakers

David T. Curiel, MD, PhD, Professor of Radiation Oncology, Director of the Biologic Therapeutics Center

Genome editing for gene therapy cures. We have developed a novel strategy to accomplish gene therapy. Our approach is based upon in vivo delivery of CRISPR/Cas9 to accomplish gene editing-based “knock-in”. Our method offers the potential to achieve gene therapy agents for a range of inherited genetic disease.

Toni Pearson, MD, Associate Professor of Pediatrics and Neurology

Gene therapy for AADC deficiency, a form of childhood-onset parkinsonism.  AADC deficiency is a rare neurometabolic disorder that presents in early childhood. Dr. Pearson will discuss current efforts to treat this disorder using targeted infusion of a gene vector (AAV2-AADC) to the midbrain in children with AADC deficiency.

On the third Thursday of every month , ICTS Precision Medicine hosts a session featuring WashU faculty working in translational genomics/precision medicine areas. Talks are typically oriented toward biotechnology, diagnostics, informatics, start-ups and precision interventions based on medical genomics innovations.