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Related Experiment Video

Updated: Jul 5, 2026

SA-β-Galactosidase-Based Screening Assay for the Identification of Senotherapeutic Drugs
07:39

SA-β-Galactosidase-Based Screening Assay for the Identification of Senotherapeutic Drugs

Published on: June 28, 2019

High-throughput screening for human galactokinase inhibitors.

Klaas J Wierenga1, Kent Lai, Peter Buchwald

  • 1Department of Pediatrics, Leonard M. Miller School of Medicine, University of Miami, Miami, Florida 33101, USA.

Journal of Biomolecular Screening
|May 21, 2008
PubMed
Summary

Researchers identified compounds that inhibit galactokinase (GALK), potentially preventing galactose toxicity in classic galactosemia. This offers a new therapeutic strategy for a serious inherited metabolic disorder.

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Area of Science:

  • Biochemistry
  • Genetics
  • Pharmacology

Background:

  • Classic galactosemia is an inherited disorder caused by galactose-1-phosphate uridyltransferase (GALT) deficiency.
  • While early diagnosis and diet prevent neonatal death, long-term complications like developmental delay and ovarian failure persist.
  • Elevated galactose-1-phosphate (gal-1-p) is a key pathogenic factor in classic galactosemia.

Purpose of the Study:

  • To investigate the hypothesis that inhibiting galactokinase (GALK) can alleviate galactose toxicity in GALT-deficient cells.
  • To identify chemical compounds that effectively inhibit human GALK activity.

Main Methods:

  • Human GALK was obtained via a bacterial expression system.
  • A high-throughput GALK assay was developed and validated (Z' factor = 0.91).

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  • A library of 50,000 chemical compounds was screened for GALK inhibitors.

    • Main Results:

    • 150 compounds inhibited GALK activity by over 86.5% at 33.3 microM in vitro.
    • A reproducibility score of at least 0.7 was achieved for confirmatory screening.
    • 34 compounds were selected for further characterization as potential therapeutic leads.

    Conclusions:

    • Inhibiting GALK is a viable strategy to reduce galactose toxicity in classic galactosemia.
    • The identified compounds show promise for developing novel therapies for this condition.
    • Further research on these 34 compounds could lead to effective treatments for classic galactosemia.