Venture Cafe – David T. Curiel, MD, PhD & Toni Pearson, MD present: Genome Editing for Gene Therapy Cures

May 16, 2019
4:00 am - 5:00 am
Venture Café St. Louis, 4240 Duncan Ave, St. Louis, MO 63110

Precision Medicine Thursdays at at Venture Café
Attendance is FREE.  Registration available at the event.


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.