enzyme replacement therapy

medicine

Written by

The Information Architects of Encyclopaedia Britannica

Last updated

July 19, 2026

Quick Summary

Enzyme replacement therapy (ERT) is a medical treatment used for certain metabolic disorders, such as adenosine deaminase (ADA) deficiency, which causes severe combined immunodeficiency (SCID). In these conditions, a specific enzyme is deficient or absent, leading to the buildup of harmful substances in the body. ERT involves administering the missing enzyme to the patient, helping to restore normal metabolic function and alleviate symptoms. For ADA deficiency, ERT is the standard treatment, though bone marrow transplantation and gene therapy are also options. It's important to note that "ERT" can also stand for estrogen replacement therapy, which is a different medical treatment.12

Summary created from Britannica articles using AI. AI can make mistakes, so verify using Britannica articles.

2 Britannica Sources

Ask Britannica AI about this topic

Britannica doesn't have a full article on this topic, so we've curated a list of our relevant coverage.

Gaucher disease
…disease may be treated by enzyme replacement therapy in which injections of imiglucerase, a synthetic analog of glucocerebrosidase that is made using recombinant DNA technology, are administered on a weekly or biweekly basis. Enzyme replacement therapy is generally ineffective in treating type 2 and type 3 Gaucher disease, because the…
Read More
…disease may be treated by enzyme replacement therapy in which injections of imiglucerase, a synthetic analog of glucocerebrosidase that is made using recombinant DNA technology, are administered on a weekly or biweekly basis. Enzyme replacement therapy is generally ineffective in treating type 2 and type 3 Gaucher disease, because the…
Read More