Chemical Therapies for Congenital Disorders of Glycosylation
العنوان: | Chemical Therapies for Congenital Disorders of Glycosylation |
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المؤلفون: | Paulina Sosicka, Bobby G. Ng, Hudson H. Freeze |
المصدر: | ACS Chem Biol |
بيانات النشر: | American Chemical Society (ACS), 2021. |
سنة النشر: | 2021 |
مصطلحات موضوعية: | congenital, hereditary, and neonatal diseases and abnormalities, Glycosylation, biology, business.industry, General Medicine, Bioinformatics, biology.organism_classification, Biochemistry, Article, chemistry.chemical_compound, Congenital Disorders of Glycosylation, chemistry, Animals, Molecular Medicine, Medicine, Dietary supplementation, business, Zebrafish |
الوصف: | Congenital disorders of glycosylation (CDG) are ultrarare, genetically and clinically heterogeneous metabolic disorders. Although the number of identified CDG is growing rapidly, there are few therapeutic options. Most treatments involve dietary supplementation with monosaccharides or other precursors. These approaches are relatively safe, but in many cases, the molecular and biochemical underpinnings are incomplete. Recent studies demonstrate that yeast, worm, fly, and zebrafish models of CDG are powerful tools in screening repurposed drugs, ushering a new avenue to search for novel therapeutic options. Here we present a perspective on compounds that are currently in use for CDG treatment or have a potential to be applied as therapeutics in the near future. |
تدمد: | 1554-8937 1554-8929 |
URL الوصول: | https://explore.openaire.eu/search/publication?articleId=doi_dedup___::5590ed3eb9c133a39772f36983e905ea https://doi.org/10.1021/acschembio.1c00601 |
حقوق: | OPEN |
رقم الأكسشن: | edsair.doi.dedup.....5590ed3eb9c133a39772f36983e905ea |
قاعدة البيانات: | OpenAIRE |
تدمد: | 15548937 15548929 |
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