Gene Therapy for Rare Neurological Disease
The Sierra Delgado Lab develops next-generation therapeutic strategies for rare and ultra-rare neurodevelopmental and neuromuscular disorders. By integrating molecular neuroscience, patient-derived cellular systems, AAV-based gene delivery and translational disease modeling, the lab aims to bridge mechanistic discovery with real-world therapeutic development for complex neurological diseases.
Lab Focus
Research Interests:
1. Molecular mechanisms underlying rare neurodevelopmental and neuromuscular disorders
2. Development of AAV-based gene therapies and translational therapeutic strategies
3. Patient-derived neuronal, astrocytic and glial disease modeling systems
4. Preclinical and IND-enabling studies for CNS and neuromuscular disorders
Areas of Expertise:
1. Translational neuroscience and rare disease therapeutic development
2. Patient-derived cellular models and in vivo disease validation
3. AAV-mediated gene delivery and preclinical therapy testing
4. Neurodevelopmental, neuromuscular, and CNS disease modeling