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Oncogene. 2019 Apr;38(16):3093-3101. doi: 10.1038/s41388-018-0625-1. Epub 2018 Dec 20.

Mitochondrial dysfunction is a key determinant of the rare disease lymphangioleiomyomatosis and provides a novel therapeutic target.

Author information

1
Department of Pharmaceutical Biotechnology, School of Pharmacy, University of Pecs, Pecs, Hungary.
2
Janos Szentagothai Research Centre, University of Pecs, Pecs, Hungary.
3
Department of Pharmaceutical Technology, School of Pharmacy, University of Pecs, Pecs, Hungary.
4
Department of Internal Medicine, School of Medicine and Clinical Centre, University of Pecs, Pecs, Hungary.
5
Department of Medical Chemistry, MTA-DE Lendulet Laboratory of Cellular Metabolism, University of Debrecen, Debrecen, Hungary.
6
Research Center for Molecular Medicine, Faculty of Medicine, University of Debrecen, Debrecen, Hungary.
7
Pulmonary, Allergy and Critical Care Division, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
8
Antoxis Ltd, Aberdeen, UK.
9
School of Medicine and Medical Sciences, University of Aberdeen, Aberdeen, UK.
10
Department of Pharmaceutical Biotechnology, School of Pharmacy, University of Pecs, Pecs, Hungary. judit.e.pongracz@gmail.com.
11
Janos Szentagothai Research Centre, University of Pecs, Pecs, Hungary. judit.e.pongracz@gmail.com.

Abstract

Lymphangioleiomyomatosis (LAM) is a rare and progressive systemic disease affecting mainly young women of childbearing age. A deterioration in lung function is driven by neoplastic growth of atypical smooth muscle-like LAM cells in the pulmonary interstitial space that leads to cystic lung destruction and spontaneous pneumothoraces. Therapeutic options for preventing disease progression are limited and often end with lung transplantation temporarily delaying an inevitable decline. To identify new therapeutic strategies for this crippling orphan disease, we have performed array based and metabolic molecular analysis on patient-derived cell lines. Our results point to the conclusion that mitochondrial biogenesis and mitochondrial dysfunction in LAM cells provide a novel target for treatment.

PMID:
30573768
PMCID:
PMC6484686
DOI:
10.1038/s41388-018-0625-1
[Indexed for MEDLINE]
Free PMC Article

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