Chemical Therapies for Congenital Disorders of Glycosylation.

Abstract

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.

• Authors: Paulina Sosicka, Bobby G Ng, Hudson H Freeze
• Journal: ACS chemical biology (ACS Chem Biol) Vol. 17 Issue 11 Pg. 2962-2971 (11 18 2022) ISSN: 1554-8937 [Electronic] United States
• PMID: 34788024 (Publication Type: Journal Article, Review, Research Support, Non-U.S. Gov't, Research Support, N.I.H., Extramural)

Topics

• Animals
• Congenital Disorders of Glycosylation (drug therapy, diagnosis, metabolism)
• Zebrafish
• Glycosylation