Innovative treatments for lysosomal diseases

Abstract

Striking therapeutic advances for lysosomal diseases have harnessed the biology of this organelle and illustrate its central rôle in the dynamic economy of the cell. Further Innovation will require improved protein-targetting or realization of therapeutic gene- and cell transfer stratagems. Rescuing function before irreversible injury, mandates a deep knowledge of clinical behaviour as well as molecular pathology – and frequently requires an understanding of neuropathology. Whether addressing primary causes, or rebalancing the effects of disordered cell function, true therapeutic innovation depends on continuing scientific exploration of the lysosome. Genuine partnerships between biotech and the patients affected by this extraordinary family of disorders continue to drive productive pharmaceutical discovery.

Keywords: chaperone; gene transfer; inhibitors of biosynthesis; innovation; lysosome; organelle; protein targeting; sphingolipids; substrate reduction; therapeutic.