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2000
Volume 21, Issue 13
  • ISSN: 1570-1808
  • E-ISSN: 1875-628X

Abstract

Introduction

Charcot Marie Tooth Disease-2 is a debilitating neurogenetic disorder that adversely affects peripheral neurons by disrupting mitochondrial activity. Mutated mitofusin-2 (MFN) is the main culprit behind disruptive mitochondrial function and is considered a therapeutic target in identifying drugs for treating this disease. This disease has no therapeutic medication except for supportive care.

Objective

The objective of the current study is to evaluate high-affinity medicinal compounds for mutated MFN-2 and describe their absorption, distribution, metabolism, excretion, and toxic attributes (ADMET).

Methods

For ADMET properties, 2,219 medicinal compounds were analyzed with AutoDock Vina using PyRX 0.9 software against MFN-2, SwissADME, and GUSAR.

Results

Results from screening studies revealed that three compounds (Liriodenine, Pinocembrin, and Vestitol) show an affinity for amino acids present in the predicted active interface of the MFN-2 protein. Moreover, these compounds render low toxicity and efficient ADME qualities, combined with blood-brain barrier permeability, drug-likeness, and lead-likeness.

Conclusion

Liriodenine, pinocembrin and vestitol are therapeutic compounds for CMT-2 treatment and should be used in further studies to confirm the results of this research.

© 2024 Bentham Science Publishers
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2025-09-09

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Discovering Drug Candidates for Charcot Marie Tooth Disease Type-2
Letters in Drug Design & Discovery 21, 2483 (2024); https://doi.org/10.2174/1570180820666230911165225
/content/journals/lddd/10.2174/1570180820666230911165225
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  • Article Type:     Letter
Keyword(s): ADME properties; charcot marie tooth disease-2; medicinal compounds; mini-peptides; mitofusin-2; virtual screening

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