Substrate reduction therapy
Substrate reduction therapy (pronounced: sub-straight re-duck-shun ther-uh-pee) is a form of treatment used primarily for lysosomal storage diseases.
Etymology
The term "substrate reduction therapy" is derived from the words "substrate", referring to the substance on which an enzyme acts, and "reduction", indicating the decrease in the amount of substrate. "Therapy" is a term used to describe a treatment intended to relieve or heal a disorder.
Overview
Substrate reduction therapy is a treatment approach that aims to reduce the accumulation of harmful substances in the body's cells. This is achieved by decreasing the production of the specific substrate that is not being properly metabolized due to a genetic enzyme deficiency.
Application
Substrate reduction therapy is used in the treatment of several lysosomal storage diseases, including Gaucher's disease, Tay-Sachs disease, and Niemann-Pick disease. These diseases are characterized by the accumulation of certain substances in the body's cells, leading to various symptoms and complications.
Related Terms
- Enzyme replacement therapy: Another form of treatment for lysosomal storage diseases, which involves replacing the deficient enzyme.
- Lysosome: A cell organelle that contains enzymes to digest particles and that is involved in the process of programmed cell death.
- Metabolism: The chemical processes in the body that convert or use energy.
- Genetic disorder: A disease caused by abnormalities in an individual's genetic material.
See Also
External links
- Medical encyclopedia article on Substrate reduction therapy
- Wikipedia's article - Substrate reduction therapy
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