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Imino sugar inhibitors for treating the lysosomal glycosphingolipidoses.

Author: ButtersTerry D, DwekRaymond A, PlattFrances M

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.

Overview

This article discussed the use of N-alkylated imino sugars as drugs for treating inherited metabolic disorders of glycosphingolipid (GSL) metabolism. These disorders are often neurodegenerative and are caused by a deficiency in catabolic enzyme activity, leading to lysosomal storage of GSL substrates. N-alkylated imino sugars have been shown to be effective inhibitors of ceramide glucosyltransferase (CGT), the key enzyme in GSL biosynthesis. This has led to the development of miglustat (Zavesca), an approved drug for treating type I Gaucher disease. The article also discussed the use of imino sugars as molecular chaperons for treating specific GSL lysosomal storage diseases. This approach aims to assist protein folding and stability of mutant enzymes.
Paper Details　
Original Abstract of the Article :
The inherited metabolic disorders of glycosphingolipid (GSL) metabolism are a relatively rare group of diseases that have diverse and often neurodegenerative phenotypes. Typically, a deficiency in catabolic enzyme activity leads to lysosomal storage of GSL substrates and in many diseases, several ot...See full text at original site
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引用元：
https://doi.org/10.1093/glycob/cwi076

データ提供：米国国立医学図書館（NLM）

Imino Sugars: A Novel Approach to Treating Lysosomal Storage Diseases

The world of [lysosomal storage diseases] is a challenging one, marked by a diverse range of debilitating conditions. This research explores the potential of [imino sugars] as a novel therapeutic strategy. It's like discovering a hidden oasis in the vast desert of genetic diseases, offering a new path to treatment.

Imino Sugars: Targeting the Root Cause of Lysosomal Storage Diseases

The authors delve into the mechanism of action of N-alkylated imino sugars as inhibitors of ceramide glucosyltransferase (CGT), a key enzyme in the biosynthesis of glycosphingolipids. The study highlights the potential of these compounds as substrate reduction therapy (SRT) and molecular chaperones. It's like finding a way to control the flow of water in a desert, redirecting it to nourish the parched landscape of cellular function.

Imino Sugars: A Promise for Future Therapies

The increasing use of imino sugars for clinical evaluation offers a glimmer of hope for treating these often intractable diseases. The study suggests that these small molecules have the potential to be orally available, penetrate the central nervous system (CNS), and have well-characterized biological and pharmacological properties. This is like finding a source of clean, fresh water in the heart of a desert, offering a lifeline to those in need.

Dr.Camel's Conclusion

This research provides a compelling case for the use of imino sugars in the treatment of lysosomal storage diseases. Their ability to act as both substrate reduction therapy (SRT) and molecular chaperones offers a promising new avenue for tackling these complex and debilitating conditions. Just as a wise traveler seeks out the best routes and resources in the desert, scientists are constantly striving to find new and effective therapies for the most challenging diseases.

Date :
  1. Date Completed 2006-01-31
  2. Date Revised 2006-11-15
Further Info :

Pubmed ID

15901676

DOI: Digital Object Identifier

10.1093/glycob/cwi076

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SNS
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in preparation
Languages

English

Positive IndicatorAn AI analysis index that serves as a benchmark for how positive the results of the study are. Note that it is a benchmark and requires careful interpretation and consideration of different perspectives.

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