BASEBiological Archive
for Science & Education
Search
HomeExploreEnzyme Replacement therapy
Therapy

Enzyme Replacement therapy

Enzyme replacement therapy

Treatment with a manufactured enzyme to replace or supplement activity that is deficient in a particular disease.

Overview

EXPLORE BY LEVEL

If a genetic condition leaves the body without enough of an , doctors may be able to give a manufactured version to replace some of that missing activity.

Quick Facts

AT A GLANCE
Explore nextMolecular medicine

Follow the Connections

See how Enzyme Replacement therapy links to other biological records.

Open network
ProcessMannose-6-phosphate Lysosomal targetingcan useProcessCrossing The blood–brain barrierlimits access ofTherapySubstrate Reduction therapycan complement
All 4 Structured Connections
Enzyme Replacement therapy · TherapyCAN USE →Mannose-6-phosphate Lysosomal targeting · Process
Crossing The blood–brain barrier · ProcessLIMITS ACCESS OF →Enzyme Replacement therapy · Therapy
Substrate Reduction therapy · TherapyCAN COMPLEMENT →Enzyme Replacement therapy · Therapy
Enzyme Replacement therapy · TherapyCAN USE →Mannose-6-phosphate Lysosomal targeting · Process

Recommended Reading

Trusted references for learning more about Enzyme Replacement therapy.

databaseGaucher diseaseMedlinePlus Genetics textbookProtein Sorting and TransportThe Cell · NCBI Bookshelf articleExploiting the diphtheria toxin internalization receptor enhances delivery of proteins to lysosomes for enzyme replacement therapyScience Advances · PMID 33310843
Research Claims

ERT can improve accessible peripheral manifestations but may not reach all affected tissues. It supplements enzyme activity rather than correcting the underlying genotype, and efficacy differs among disorders and organs.