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Molecular medicines for the lysosome

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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.

Critical functions of lysosomes are revealed by the astonishing clinical diversity of genetic 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.

Lysosomes may be accessed by proteins delivered at the cell surface and the spectacular success of macrophage-targeted enzyme therapy for Gaucher disease has stimulated development of inhibitors that rebalance defective lysosomal substrate recycling. The diseased brain remains a formidable challenge but gene therapy is promising and in early clinical development.

This talk is part of the Cambridge Philosophical Society series.

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