Substrate reduction therapy offers an approach to treatment of certain metabolic disorders, especially glycogen storage diseases and lysosomal storage disorders. In a storage disorder, a critical failure in a metabolic pathway prevents cellular breakdown and disposal of some large molecule. If residual breakdown through other pathways is insufficient to prevent harmful accumulation, the molecule accumulates in the cell and eventually interferes with normal biological processes. Examples of lysosomal storage disorders include Gaucher's disease, Tay–Sachs disease, Sandhoff disease, and Sanfilippo syndrome.
In a metabolic or genetic pathway, enzymes catalyze a series of reactions. Each enzyme is regulated or mediated by one gene through its RNA and protein products. At each phase in the pathway, enzyme activity catalyzes a reaction in which a precursor molecule (the substrate) is transformed into its next intermediate state. Failure of the metabolic pathway leads to accumulation of the substrate, with possible harmful effects. Substrate reduction therapy addresses this failure by reducing the level of the substrate to a point where residual degradative activity is sufficient to prevent substrate accumulation.
and 21 Related for: Substrate reduction therapy information
Substratereductiontherapy offers an approach to treatment of certain metabolic disorders, especially glycogen storage diseases and lysosomal storage...
therapy, enzyme replacement therapy (ERT), substratereductiontherapy (SRT), and potentially enzyme enhancement therapy (EET). In addition to the clinical...
therapy: Replacement of the missing enzyme to clear the lipids (GL-3) from the cells Substrate synthesis inhibition, also called substratereduction therapy:...
article in Journal of the American Medical Association the oral substratereductiontherapy resulted in "significant improvements in spleen volume, hemoglobin...
specialized centers for a number of these diseases. In addition, substratereductiontherapy, a method used to decrease the production of storage material...
Weinreb, NJ; Cox, TM (27 March 2015). "Enzyme replacement and substratereductiontherapy for Gaucher disease". The Cochrane Database of Systematic Reviews...
The molecules upon which enzymes may act are called substrates, and the enzyme converts the substrates into different molecules known as products. Almost...
that builds up when the enzyme doesn't work; this is called substratereductiontherapy. Miglustat is an iminosugar, a synthetic analogue of D-glucose...
they will fit into the enzyme's active site and compete with the native substrate (HMG-CoA). This competition reduces the rate by which HMG-CoA reductase...
Feminizing hormone therapy, also known as transfeminine hormone therapy, is hormone therapy and sex reassignment therapy to change the secondary sex characteristics...
Cholesterol regulates the biological process of substrate presentation and the enzymes that use substrate presentation as a mechanism of their activation...
(80 mg) atorvastatin therapy is more effective than regular dose or low dose statin therapy at preventing CI-AKI. Atorvastatin therapy can also help to prevent...
Photodynamic therapy (PDT) is a form of phototherapy involving light and a photosensitizing chemical substance used in conjunction with molecular oxygen...
therapy using methadone or buprenorphine. Such treatment reduces the risk of death. Additionally, they may benefit from cognitive behavioral therapy,...
ferrous to ferric conversion to generate a tyrosyl free radical. Reduction of NDP substrates occurs under aerobic conditions. Class I reductases are divided...
agonist of the androgen receptor (AR). It is not a substrate for 5α-reductase and is a poor substrate for 3α-hydroxysteroid dehydrogenase (3α-HSD), and...
risk of cardiovascular disease. Solbinsiran is a GalNAc-conjugated Dicer-substrate siRNA (DsiRNA) that targets ANGPTL3 expression in the liver. ANGPTL3 plays...
sometimes they donate an electron to the substrate, sometimes they abstract a hydrogen atom from the substrate. At the end of this process, the photosensitizer...
spiritual-based approach to treating addiction. Therapy typically involves the use of cognitive-behavioral therapy, an approach that looks at the relationship...
Insulin receptor substrate 1 (IRS-1) is a signaling adapter protein that in humans is encoded by the IRS1 gene. It is a 131 kDa protein with amino acid...
Global Information