Vidéo décrivant le projet du Dr Nevan Krogan “Discovery of new targets for therapeutic interventions in ARSACS disease”

“The development of novel treatments, through the discovery of new drugs or the repurposing of FDA-approved drugs and INDs, requires a detailed understanding of the biology of disorders. Protein complexes and networks work together to ensure healthy cell physiology and are altered in disease. Uncovering how disease mutations alter protein networks opens the door for novel target identification and allows the investigation of how drugs can restore a healthy state. To rapidly identify new protein targets for therapeutic interventions for ARSACS disease, we will apply unbiased mass spectrometry-based proteomics and innovative computational approaches to globally uncover the cellular signaling pathways and kinases affected by deletion of the SACS gene. We will use this information to identify drugs acting on the protein targets in these pathways and test them in cell culture assays. While we plan to identify both novel as well as existing drugs for therapeutic benefit, we will prioritize FDA-approved compounds as well as new investigational drugs (INDs). This “drug repurposing” strategy provides an expedited path from discovery to translational impact for this debilitating and poorly-studied condition. Once promising drug candidates have been identified, subsequent studies can be designed to test candidates in animal models and human clinical trials”.

Financement: 151,520$ USD

Le projet de recherche est financé en partenariat avec la Fondation de l’UCSF, Laboratoire Krogan en l’honneur d’Allison Trainor.

Durée: un an


Dr. Nevan Krogan, Cellular Molecular Pharmacology School of Medicine, University of California San Francisco
1700 4th Street,San Francisco, California, CA 94158

Tel: 415 476 2980