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The distribution of the therapeutic enzyme in the body (biodistribution) after these IV infusions is not uniform. [10] The enzyme in less available to certain areas in the body, like the bones, lungs, brain. For this reason, many symptoms of lysosomal storage diseases remain untreated by ERT, especially neurological symptoms. [10]
In clinical studies, oral administration of proteolytic enzymes to healthy volunteers resulted in immunomodulatory effects and systemic therapy before and after exhaustive exercise increased maximal concentric strength, and had favorable effects on inflammatory, metabolic and immune biomarkers.
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 ...
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An example target for targeted therapy is the BCR-ABL1 protein produced from the Philadelphia chromosome, a genetic lesion found commonly in chronic myelogenous leukemia and in some patients with acute lymphoblastic leukemia. This fusion protein has enzyme activity that can be inhibited by imatinib, a small molecule drug. [152] [153] [154] [155]
Systemic enzyme therapy (consisting of combinations of proteolytic enzymes such as bromelain, trypsin, chymotrypsin, and papain) has been investigated in Europe to evaluate the efficacy in breast, colorectal, and plasmacytoma cancer patients.
Treatment types can be classified into local therapy (surgery and radiotherapy) and systemic treatment (chemo-, endocrine, and targeted therapies). Local therapy is most efficacious in early stage breast cancer, while systemic therapy is generally justified in advanced and metastatic disease, or in diseases with specific phenotypes.
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 ...
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