The Niemann-Pick type C lysosomal pathway as a novel hub in host: pathogen interactions


  • Prof Frances Platt

    University of Oxford

Project summary

Studying rare diseases can bring to light disease mechanisms that occur in more common diseases. For many years we have been studying a rare neurodegenerative disease called Niemann-Pick type C (NPC) disease. We have unexpectedly found that the faulty protein in this disease is targeted by the bacterium that cause tuberculosis (TB). TB inhibits the function of this protein so it can prevent itself being killed by the cell it infects. This unexpected link between NPC and TB now gives us new methods for treating TB based on treatments we have developed for NPC. The Ebola virus also uses this very same protein to infect cells which suggests that the pathway in NPC disease is a hub that is targeted by multiple infectious diseases.  

This study aims to understand how infectious agents manipulate this pathway and use this knowledge to develop a new generation of antibiotics and antiviral drugs.