- RNA regulation and disease
- Virus-based gene therapy research
- RNA and protein synthesis mechanisms
- interferon and immune responses
- CRISPR and Genetic Engineering
- RNA Interference and Gene Delivery
- RNA Research and Splicing
- Cancer-related molecular mechanisms research
The Ohio State University
2024
Nationwide Children's Hospital
2023-2024
University of California, Davis
2024
Office of Infectious Diseases
2024
OBJECTIVES/GOALS: Vanishing White Matter Disease (VWM), is a childhood neurodegenerative leukodystrophy that presents with motor deficits, neurologic decline, and seizures leading to death.There are no treatments. Herein we investigate adeno-associated virus serotype 9 (AAV9) gene addition therapy for VWM. METHODS/STUDY POPULATION: To serve as baseline disease correction, characterized the severe VWM Eif2b5 I98M murine model clinically relevant readouts including function, gait mapping...