PROTEOMIC APPROACHES FOR THE DEVELOPMENT OF NOVEL THERAPEUTIC STRATEGIES IN AMYOTROPHIC LATERAL SCLEROSIS

Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease for which there is no therapy. Proteomics is a group of sophisticated technologies which are able to identify and quantify a large number of proteins at the same time in tissues and biological fluids with high sensitivity and accuracy. Proteins are the effectors of almost all cellular functions and alteration of their levels or structure is at the basis of several diseases. Therefore proteins are the major targets of the pharmacological treatments. Identifying the alteration of proteins in a disease state is at the basis of developing effective drugs. We plan to apply proteomic technologies to the different stages of drug development, in cellular and animal models of the pathology and in samples (blood cells, cerebrospinal fluid and fibroblasts) of ALS patients. In this way, we will identify a panel of proteins which are potential targets of pharmacological treatments and candidate biomarkers of disease for early diagnosis, monitoring the progression of the disease and eventually the effects of drugs. We believe that these studies will contribute to accelerate the process toward the identification of efficient therapeutic strategies in ALS.