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Related Concept Videos

Phase I Reactions: Reductive Reactions01:27

Phase I Reactions: Reductive Reactions

Phase I biotransformation reductive reactions are chemical processes that modify drugs by introducing or revealing polar functional groups via reduction. Enzymes called reductases catalyze these reactions, playing a pivotal role in drug metabolism by transforming lipophilic drugs into more polar, water-soluble metabolites for easy excretion. An essential type of reductive reaction is the carbonyl group reduction, where aldehydes and ketones are reduced to alcohols. An example is the...
Sulfur Assimilation01:20

Sulfur Assimilation

Sulfur is an essential element in biological systems, contributing to synthesizing key biomolecules, including amino acids such as cysteine and methionine, and cofactors such as coenzyme A and biotin. Microorganisms primarily assimilate sulfur as sulfate (SO₄²⁻) from the environment, which must undergo a series of biochemical transformations before it can be incorporated into cellular components. As sulfate is highly oxidized, it must undergo assimilatory sulfate reduction to become...
Antiprotozoal Agents01:21

Antiprotozoal Agents

Leishmaniasis is a widespread parasitic disease caused by several Leishmania species. It affects millions of people each year and remains a major public health problem in endemic regions. First-line treatment relies on pentavalent antimonials, including meglumine antimoniate and sodium stibogluconate. Even so, how these drugs work has not been fully clear, especially their interaction with parasite-specific biochemical pathways. One key target is trypanothione reductase (TR), an enzyme that...
Alcohols from Carbonyl Compounds: Reduction02:23

Alcohols from Carbonyl Compounds: Reduction

Reduction is a simple strategy to convert a carbonyl group to a hydroxyl group. The three major pathways to reduce carbonyls to alcohols are catalytic hydrogenation, hydride reduction, and borane reduction.
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Phase II Reactions: Miscellaneous Conjugation Reactions01:19

Phase II Reactions: Miscellaneous Conjugation Reactions

Phase II biotransformations are detoxification mechanisms that conjugate xenobiotics with endogenous substances, neutralizing their toxicity.
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Redox Reactions

Redox reactions are vital biochemical processes that underpin energy metabolism in cells. These reactions involve the transfer of electrons between molecules, occurring in tandem as oxidation and reduction. Oxidation refers to the loss of electrons, while reduction denotes their gain. This coupling ensures the seamless flow of electrons through metabolic pathways. For example, in bacterial metabolism, glucose undergoes oxidation to carbon dioxide, while oxygen is simultaneously reduced to...

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Related Experiment Video

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Development of Sulfidogenic Sludge from Marine Sediments and Trichloroethylene Reduction in an Upflow Anaerobic Sludge Blanket Reactor
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Published on: October 15, 2015

Substrate reduction therapy.

Frances M Platt1, Mylvaganam Jeyakumar

  • 1Department of Pharmacology, University of Oxford, Mansfield Road, Oxford, UK. frances.platt@pharm.ox.ac.uk

Acta Paediatrica (Oslo, Norway : 1992)
|May 28, 2008
PubMed
Summary
This summary is machine-generated.

Substrate reduction therapy (SRT) offers an oral treatment for type 1 Gaucher disease, potentially benefiting central nervous system (CNS) conditions. SRT may also be a future component of combination therapies for lysosomal storage diseases (LSDs).

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Published on: August 13, 2021

Area of Science:

  • Biochemistry
  • Pharmacology
  • Genetics

Background:

  • Lysosomal storage diseases (LSDs) present significant therapeutic challenges, particularly for central nervous system (CNS) and peripheral manifestations.
  • Type 1 Gaucher disease has two main treatments: enzyme replacement therapy (ERT) and substrate reduction therapy (SRT).

Purpose of the Study:

  • To review the current status of substrate reduction therapy (SRT) for treating Gaucher disease and other LSDs.
  • To discuss the potential of SRT, including orally available small molecules, for managing LSDs with CNS pathology.

Main Methods:

  • Review of preclinical and clinical studies on substrate reduction therapy (SRT).
  • Discussion of the mechanism of action for both enzyme replacement therapy (ERT) and substrate reduction therapy (SRT).

Main Results:

  • Substrate reduction therapy (SRT) provides an oral alternative to ERT for type 1 Gaucher disease.
  • SRT, specifically miglustat, crosses the blood-brain barrier, offering potential for LSDs with CNS involvement.
  • SRT shows promise in managing glycosphingolipid storage in the brain, as suggested by Niemann-Pick disease type C studies.

Conclusions:

  • SRT is a viable oral treatment option for type 1 Gaucher disease patients unable or unwilling to undergo ERT.
  • Miglustat (SRT) may play a role in managing CNS manifestations of LSDs, including glycosphingolipid storage in the brain.
  • SRT's synergistic potential positions it as a key component in future combination therapies for LSDs.