From rare to common and back again: 60 years of lysosomal dysfunction

  • Coutinho M
  • Alves S
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Sixty years after its discovery, the lysosome is no longer considered as cell's waste bin but as an organelle playing a central role in cell metabolism. Besides its well known association with lysosomal storage disorders (mostly rare and life-threatening diseases), recent data have shown that the lysosome is also a player in some of the most common conditions of our time; and, perhaps even most important, it is not only a target for orphan drugs (rare disease therapeutic approaches) but also a putative target to treat patients suffering from common complex diseases worldwide. Here we review the striking associations linking rare lysosomal storage disorders such as the well-known Gaucher disease, or even the recently discovered, extremely rare Neuronal Ceroid Lipofuscinosis-11 and some of the most frequent, multifaceted and complex disorders of modern society such as cancer, Parkinson's disease and frontotemporal lobar degeneration.

Author-supplied keywords

  • Cancer
  • Frontotemporal lobar degeneration (FTLD)
  • Gaucher disease (GD)
  • Lysosomal storage disorders (LSDs)
  • Neuronal ceroid lipofuscinosis (NCL)
  • Parkinson's disease (PD)

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