Therapy
Substrate Reduction therapy
Substrate reduction therapy
A strategy that lowers production of a material that accumulates when its breakdown pathway is impaired.
Overview
EXPLORE BY LEVELIf a cell cannot break down a material properly, one approach is to make less of that material. This may reduce how much builds up.
Quick Facts
AT A GLANCEExplore nextMolecular medicine
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Recommended Reading
Trusted references for learning more about Substrate Reduction therapy.
Research Claims
SRT response depends on substrate synthesis, residual degradation, tissue exposure, disease stage, and off-target effects. It is mechanistically distinct from enzyme replacement and gene correction.