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  2. Enzyme replacement therapy - Wikipedia

    en.wikipedia.org/wiki/Enzyme_replacement_therapy

    The accumulated compounds are responsible for the symptoms of these disorders, and they form via a multi-step biological pathway. [10] Substrate reduction therapy uses a small molecule to interrupt this multi-step pathway and inhibit the biosynthesis of these compounds. [10] This type of treatment is taken orally. [10]

  3. Multienzyme complex - Wikipedia

    en.wikipedia.org/wiki/Multienzyme_complex

    In molecular biology, a multienzyme complex is a protein complex containing several copies of one or more enzymes packed into one macromolecular assembly. Multienzyme complexes carry out a single or multi-step biochemical reaction taking place within cells. It allows the cell to segregate certain biochemical pathways into one place in the cell. [1]

  4. Cipaglucosidase alfa - Wikipedia

    en.wikipedia.org/wiki/Cipaglucosidase_alfa

    [4] [5] Cipaglucosidase alfa is a recombinant human acid α-glucosidase enzyme replacement therapy that provides an exogenous source of acid α-glucosidase. [ 5 ] The most common side effects include chills, dizziness, flushing, sleepiness, chest discomfort, cough, swelling at the infusion site and pain. [ 5 ]

  5. DEPT (medicine) - Wikipedia

    en.wikipedia.org/wiki/DEPT_(medicine)

    GDEPT is a suicide gene therapy in which the enzyme required for prodrug conversion is produced within the target cell, using a gene delivered to it by gene therapy. When an adequate differential exists between the targeted cell and endogenous tissue, non-toxic prodrug is administered and is subsequently converted into its toxic form within the ...

  6. Serine hydroxymethyltransferase - Wikipedia

    en.wikipedia.org/wiki/Serine_hydroxymethyl...

    PyMol rendered crystal structure of serine hydroxymethyltransferase. Serine hydroxymethyltransferase (SHMT) is a pyridoxal phosphate (PLP) (Vitamin B 6) dependent enzyme (EC 2.1.2.1) which plays an important role in cellular one-carbon pathways by catalyzing the reversible, simultaneous conversions of L-serine to glycine and tetrahydrofolate (THF) to 5,10-methylenetetrahydrofolate (5,10-CH 2 ...

  7. List of enzymes - Wikipedia

    en.wikipedia.org/wiki/List_of_enzymes

    Function: Amylase is an enzyme that is responsible for the breaking of the bonds in starches, polysaccharides, and complex carbohydrates to be turned into simple sugars that will be easier to absorb. Clinical Significance: Amylase also has medical history in the use of Pancreatic Enzyme Replacement Therapy (PERT). One of the components is ...

  8. Thiopurine methyltransferase - Wikipedia

    en.wikipedia.org/wiki/Thiopurine_methyltransferase

    7172 22017 Ensembl ENSG00000137364 ENSMUSG00000021376 UniProt P51580 O55060 RefSeq (mRNA) NM_000367 NM_001346817 NM_001346818 NM_016785 RefSeq (protein) NP_000358 NP_001333746 NP_001333747 NP_058065 Location (UCSC) Chr 6: 18.13 – 18.16 Mb Chr 13: 47.18 – 47.2 Mb PubMed search Wikidata View/Edit Human View/Edit Mouse Thiopurine methyltransferase or thiopurine S-methyltransferase (TPMT) is ...

  9. Sphingolipidoses - Wikipedia

    en.wikipedia.org/wiki/Sphingolipidoses

    Enzyme replacement therapy is available to treat mainly Fabry disease and Gaucher disease, and people with these types of sphingolipidoses may live well into adulthood. The other types are generally fatal by age 1 to 5 years for infantile forms, but progression may be mild for juvenile- or adult-onset forms.