Research Description:

Our research focuses on the development of gene and cell-based therapies for the treatment of metabolic diseases of the liver that commonly result in the development of intellectual disabilities in children. We have two active research directions:
1. Development of gene-based (adeno-associated virus, mRNA) therapies to restore nitrogen metabolism and ureagenesis.
2. Exploring the underlying neurobiology in arginase deficiency to gain a greater understanding of the neuropathology that exists in this enzyme deficiency.

  • Faculty member at UCLA, 2004 – present
  • Postdoctoral Training, UCSF