The core research focus of the Gray Lab is to develop adeno-associated virus (AAV) gene transfer vector systems for clinically-relevant global gene transfer to the central and peripheral nervous system.  This research focus has also included preclinical studies to apply these engineered AAV systems toward treatments for neurological diseases in animal models.  Currently these include:

  • Rett Syndrome
  • Giant Axonal Neuropathy (GAN)
  • Multiple Sulfatase Deficiency
  • SURF1-related Leigh Syndrome
  • SLC6A1
  • Tay-Sachs
  • Sandhoff
  • Krabbe
  • Aspartylglucosaminuria (AGU)
  • Charcot-Marie Tooth
  • Batten Diseases
  • GNAO1 Deficiency
  • DDX3X Deficiency
  • Monoamine Oxidase Deficiency
  • Angelman Syndrome

Research has expanded into human clinical studies to test a gene therapy approach for GAN.

Future directions for the lab include:

  1. Continued development and optimization of AAV vectors specifically tailored toward CNS applications
  2. Expanding this program to include therapeutic approaches for other applicable CNS disorders
  3. Facilitating the translation of these approaches from bench to clinic

Must See Videos