Enzyme replacement therapy (ERT) is a medical treatment which replaces an enzyme that is deficient or absent in the body.[1] Usually, this is done by giving the patient an intravenous (IV) infusion of a solution containing the enzyme.[1]
ERT is available for some lysosomal storage diseases: Gaucher disease, Fabry disease, MPS I, MPS II (Hunter syndrome), MPS VI and Pompe disease.[1] ERT does not correct the underlying genetic defect, but it increases the concentration of the enzyme that the patient is lacking.[1] ERT has also been used to treat patients with severe combined immunodeficiency (SCID) resulting from an adenosine deaminase deficiency (ADA-SCID).[2]
Other treatment options for patients with enzyme or protein deficiencies include substrate reduction therapy, gene therapy, and bone-marrow derived stem cell transplantation.[1][3][4]
^ abcdeRies, M (2017). "Enzyme replacement therapy and beyond-in memoriam Roscoe O. Brady, M.D. (1923–2016)". Journal of Inherited Metabolic Disease. 40 (3): 343–356. doi:10.1007/s10545-017-0032-8. PMID 28314976. S2CID 31320877.
^Booth, C; Hershfield, M; Notarangelo, L; Buckley, R; Hoenig, M; Mahlaoui, N; Cavazzana-Calvo, M; Aiuti, A; Gaspar, H. B. (2007). "Management options for adenosine deaminase deficiency; proceedings of the EBMT satellite workshop (Hamburg, March 2006)". Clinical Immunology. 123 (2): 139–47. doi:10.1016/j.clim.2006.12.009. PMID 17300989.
^Ries, M (2017). "Enzyme replacement therapy and beyond-in memoriam Roscoe O. Brady, M.D. (1923–2016)". Journal of Inherited Metabolic Disease. 40 (3): 343–356. doi:10.1007/s10545-017-0032-8. PMID 28314976. S2CID 31320877. [verification needed]
^Ries, M (2017). "Enzyme replacement therapy and beyond-in memoriam Roscoe O. Brady, M.D. (1923–2016)". Journal of Inherited Metabolic Disease. 40 (3): 343–356. doi:10.1007/s10545-017-0032-8. PMID 28314976. S2CID 31320877. [verification needed]
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