Positions

Associate Professor
Molecular and Cellular Biology
Center for Cell and Gene Therapy
Baylor College of Medicine
Member
Texas Medical Center Digestive Diseases Center (DDC)
Dan L Duncan Comprehensive Cancer Center (DLDCCC)
Baylor College of Medicine
Houston, Texas, United States

Education

MD from School of Medicine, University of Bern
PhD from School of Medicine, University of Bern
Clinical Fellowship at University Hospital Bern
Internal Medicine and Gastroenterology
Postdoctoral Fellowship at Salk Institute for Biological Studies

Professional Interests

  • Genome engineering in the liver
  • Metabolic liver disease
  • Human liver chimeric mice
  • Liver cancer
  • Hepatitis B virus
  • Cell based therapy for liver disease

Professional Statement

We are interested in translating basic science breakthroughs into therapeutic approaches for liver disorders. Our research is therefore best described as from “bench to bedside”. We are not focused on one liver disorder in particular but our interest ranges from liver cancer to viral hepatitis to metabolic liver disorders.

Basic science is often poorly translated into the clinic due to lack of experimental animal models for human disease. Indeed, metabolic pathways differ significantly across the animal kingdom. Therefore, we spend considerable effort generating new in vivo systems for primary and diseased human hepatocytes.

Our therapeutic strategies include new concepts and biologics. The advent of CRISPR/Cas9 technology has generated enormous excitement throughout the many fields of biology and biomedical research, though its broad therapeutic application has been stymied by lack of a feasible approach for genome editing that obviates its chief risks. We recently developed a therapeutic genome editing approach, called metabolic pathway reprogramming, that couples the power of CRISPR/Cas9 technology with a strategy from pharmacology, namely, inhibition of an enzymatic pathway rather than direct editing of a disease-causing gene. We have demonstrated the efficacy of this approach using CRISPR/Cas9 to convert the fatal type I tyrosinemia into benign type III tyrosinemia in mice.

Selected Publications