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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 ...
An early collaboration with Norman Radin focused on substrate reduction as an alternative to enzyme replacement therapy for the treatment of lysosomal disorders such as Gaucher disease. It was suggested that substrate reduction posits that inhibition of metabolites that accumulate in the lysosome due to the loss of activity of a specific ...
Substrate reduction therapy is FDA approved and there is at least one treatment available on the market. [10] Gene therapy aims to replace a missing protein in the body through the use of vectors, usually viral vectors. [11] In gene therapy, a gene encoding for a certain protein is inserted into a vector. [11]
A gene therapy treatment that is in early-phase clinical trials, [40] [41] with the technology licensed to AvroBio. [42] The substrate reduction therapy Venglustat (Ibiglustat) under development by Sanofi-Genzyme [43] Bio-better ERT (CDX-6311) under pre-clinical development by the company Codexis [citation needed]
Several therapy options are currently being investigated using clinical trials primarily in late infantile patients. These therapies include gene therapy, enzyme replacement therapy (ERT), substrate reduction therapy (SRT), and potentially enzyme enhancement therapy (EET). In addition to the clinical trials, there are several other pre-clinical ...
Miglustat is indicated to treat adults with mild to moderate type I Gaucher disease for whom enzyme replacement therapy is unsuitable. [14]In the European Union, miglustat (Opfolda), in combination with cipaglucosidase alfa, is a long-term enzyme replacement therapy in adults with late-onset Pompe disease (acid α‑glucosidase [GAA] deficiency).
Substrate reduction therapy (SRT) is also currently being studied to treat Sanfilippo syndrome. Since Sanfilippo syndrome occurs due to the body's inability to break down GAGs, SRT functions by targeting the genes responsible for the synthesis of GAGs to restore the balance of production and breakdown of GAGs. [ 15 ]
Substrate reduction therapy; Supercow (dairy) T. Therapeutic gene modulation; Transfection; V. Vectors in gene therapy This page was last edited on 29 August 2020, at ...