Pyramidal neurons in mouse cerebral cortex transduced with AAV. The Bailey Lab investigates the use of adeno-associated viral (AAV) vectors for therapeutic gene delivery for neurological disorders.
We work on monogenetic pediatric disorders, including SLC13A5 epileptic encephalopathy, multiple sulfatase deficiency, Charcot Marie Tooth disease type 4J, giant axonal neuropathy, and ECHS1 deficiency.
We are also developing approaches that bring AAV to bear on more complex, non-inherited neurodegenerative diseases.
Our lab uses gene-replacement and gene-silencing approaches to optimize AAV vector delivery to the nervous system and facilitates the translation of these therapies into human testing. This work requires the use of cellular and rodent models to investigate disease pathogenesis, improve vector efficacy, and identify biomarkers for human testing. Our main research interests are:
- Engineering AAV vectors
- Developing gene therapies for genetic pediatric disorders
- Developing gene therapies for neurodegenerative diseases
- Selective targeting of AAV vectors to the central and peripheral nervous systems
- Translating gene therapies to clinical trials