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Treating lysosomal storage disorders: current practice and future prospects

Frances M Platt1, Robin H Lachmann

  • 1Department of Pharmacology, University of Oxford, Mansfield Road, Oxford, OX1 3QT, UK. frances.platt@pharm.ox.ac.uk

Biochimica Et Biophysica Acta
|October 1, 2008
PubMed

Insights

Lysosomal storage disorders have seen therapeutic advances, but treating central nervous system effects remains a major challenge. This review examines current therapies and their associated scientific, medical, social, and ethical considerations.

Area of Science:

  • Biochemistry
  • Genetics
  • Pharmacology

Background:

  • Over 40 human diseases stem from impaired lysosomal function.
  • Lysosomal storage disorders (LSDs) are a group of genetic conditions.
  • Lysosomes are vital cellular organelles responsible for waste breakdown.

Purpose of the Study:

  • To review current and emerging therapies for lysosomal storage disorders.
  • To discuss the challenges in treating central nervous system (CNS) manifestations of LSDs.
  • To explore the scientific, medical, social, and ethical implications of LSD therapies.

Main Methods:

  • Literature review of existing research and clinical trials.
  • Analysis of therapeutic strategies for LSDs.
  • Discussion of scientific, medical, and ethical considerations.

Main Results:

  • Significant progress has been made in developing therapies for many LSDs.
  • Several therapies are in clinical use or trials.
  • Effective treatment for CNS manifestations of LSDs remains a significant hurdle.

Conclusions:

  • Therapeutic development for LSDs has advanced considerably.
  • Addressing CNS involvement in LSDs requires innovative approaches.
  • A multidisciplinary perspective is crucial for evaluating LSD therapies.

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