Gary Bassell, PhD

The research interests of our laboratory are to understand the diverse and critical roles played by mRNA binding proteins and associated factors in the posttranscriptional regulation of gene expression in the nervous system, and investigate how these processes go awry in neurodevelopmental and neurodegenerative disorders. We investigate the normal mechanism, function and regulation of mRNA binding proteins in mRNA transport and local protein synthesis needed for neuronal development and synaptic plasticity. We investigate pathomechanisms for Fragile X syndrome (FXS), myotonic dystrophy (DM), amyotrophic lateral sclerosis and frontotemporal dementia (ALS/FTD). We are using mouse models of neurological diseases to assess the function of mRNA regulation and local protein synthesis in axon guidance, synapse development and neuronal signaling.

Efforts are also underway to evaluate different therapeutic modalities in mouse and human stem cell derived models of neurological diseases. Our research utilizes an integrated multi-disciplinary approach that involves cellular, molecular, biochemical, physiological, and behavioral methods and paradigms. These studies are expected to reveal new mechanisms important for neuronal development and function, and targeted approaches for therapeutic intervention that treat underlying molecular defects in SMA, ALS/FTD, myotonic dystrophy, Fragile X syndrome and autism spectrum disorders.