Rare Diseases

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In June 2024, NIH published RFA-HD-25-002 : Centers for Collaborative Research in Fragile X and FMR1-Associated Conditions (P50 Clinical Trial Optional).

Please note: In July 2024, NIH issued NOT-HD-24-026 : Notice to Extend the Application Receipt Date for RFA-HD-25-002, which extends the Request for Applications (RFA) deadline to August 20, 2024.

Weidong Wang , PhD, Chief

Bryan J. Traynor , M.D., Ph.D., Chief

The Neuromuscular Diseases Research Section investigates the causes and biology of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). Our focus is on turning discoveries into better diagnosis and faster development of treatments. We combine large-scale human genetics with detailed molecular profiling to understand disease processes, improve patient grouping, and create biomarkers and outcome tools for clinical trials.

We strive to understand fundamental relationships between function and structure in living tissues, primarily in neural tissue and in extracellular matrix (ECM). Specifically, we are interested in how microstructure, hierarchical organization, composition, and material properties all affect biological function and dysfunction. We investigate biological and physical model systems, such as “engineered” tissue constructs and tissue analogs at different time and length scales.

The Hoffman group combines molecular, biochemical and electrophysiological approaches to investigate the roles of dendritic voltage-gated and neurotransmitter-activated channels in regulating neuronal development and synaptic plasticity in the mammalian hippocampus. To achieve this, the lab comprises an interdisciplinary team with expertise in cellular and molecular biology, biochemistry, electrophysiology, fluorescence imaging and computer modeling. These approaches allow us to assay channel function and regulation at the molecular scale in living cells.