Therapy

Substrate Reduction therapy

Substrate reduction therapy

A strategy that lowers production of a material that accumulates when its breakdown pathway is impaired.

Overview

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If 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 GLANCE
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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.