Drug Repurposing Reveals mTOR Inhibition as a Promising Strategy for Epidermolysis Bullosa Simplex.
Ragot H,Hovnanian A
Source :
J Invest Dermatol
2021 Dec 17
Pmid / DOI:
34924185
Abstract
Drug repurposing has the potential to discover new treatments for diseases with high unmet medical needs. Lee et al. (2021) combined transcriptomics and computational analysis of drug-target databases to identify novel therapies for epidermolysis bullosa simplex. Differential gene expression analysis of blister epidermis identified the phosphoinositide 3-kinase/protein kinase B/mTOR signaling pathway as central. A pilot study using a topical mTOR inhibitor showed marked improvement.Copyright © 2021 The Authors. Published by Elsevier Inc. All rights reserved.