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 (in alphabetic order):
- Angelman Syndrome *
- Aspartylglucosaminuria (AGU)
- Charcot-Marie Tooth Disease Type 4A
- Charcot-Marie Tooth Disease Type 4J
- CLN1 Batten Diseases
- CLN5 Batten Diseases *
- CLN7 Batten Diseases
- DHPS gene deficiency
- DDX3X Deficiency
- Giant Axonal Neuropathy (GAN)
- Krabbe *
- LNPK Deficiency
- Monoamine Oxidase Deficiency
- MPS IIIC
- Multiple Sulfatase Deficiency
- Pitt-Hopkins syndrome *
- Pyruvate Dehydrogenase Complex Deficiency (PDHA1 gene)
- Rett Syndrome *
- Sandhoff *
- Spastic Paraplegia Type 49 (SPG49, TECPR2 gene deficiency)
- Spastic Paraplegia Type 50 (SPG50, AP4M1 gene deficiency)
- SLC6A1
- SURF1-related Leigh Syndrome
- Tay-Sachs *
* currently only supporting through collaborations
Research has expanded into human clinical studies to test a gene therapy approach for GAN, CLN1 Batten disease, CLN5 Batten disease, CLN7 Batten disease, SPG50, Sandhoff disease, Tay-Sachs disease, and Rett Syndrome.
Future directions for the lab include:
- Continued development and optimization of AAV vectors specifically tailored toward CNS applications
- Expanding this program to include therapeutic approaches for other applicable CNS disorders
- Facilitating the translation of these approaches from bench to clinic