Amyotrophic lateral sclerosis
Several distinct molecular mechanisms may play a role in motor neuron degeneration. In approximately 10% of patients, amytrophic lateral sclerosis (ALS) occurs in families with an autosomal dominant mode of inheritance caused by mutations in genes that appear functionally heterogeneous. Sporadic ALS is considered a complex multifactorial disease with interplay of genetic and environmental factors affecting susceptibility and clinical expression.
The goal of our research is to develop effective treatment for patients with ALS, by delineating molecular pathways that drive motor neuron degeration using data and samples obtained from patients, transgenic ALS mice and neuronal cell culture models. To reach this goal we use several approaches which are described here.