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SUMMARY:Molecular medicines for the lysosome - Professor Timothy Cox MD FR
 CP FMedSci\, Professor of Medicine Emeritus
DTSTART:20161107T180000Z
DTEND:20161107T190000Z
UID:TALK67226@talks.cam.ac.uk
CONTACT:Beverley Larner
DESCRIPTION:Abstract: This year saw the award of the third Nobel prize for
  research into the cell biology of the lysosome: an organelle for defence\
 , recycling and energy-sensing of the metabolic economy.\n\nCritical funct
 ions of lysosomes are revealed by the astonishing clinical diversity of ge
 netic defects in this organelle – many of these rare diseases affect the
  brain. We are learning much about common conditions\, such as myeloma and
  Parkinson disease\, from their study.\n\nLysosomes may be accessed by pro
 teins delivered at the cell surface and the spectacular success of macroph
 age-targeted enzyme therapy for Gaucher disease has stimulated development
  of  inhibitors that rebalance defective lysosomal substrate recycling. Th
 e diseased brain remains a formidable challenge but gene therapy is promis
 ing and in early clinical development.\n\n 
LOCATION:Bristol-Myers Squibb Lecture Theatre\, Department of Chemistry
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