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

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 destructi...

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Bibliographic Details
Published in:Oncogene 2019-04, Vol.38 (16), p.3093-3101
Main Authors: Abdelwahab, E M M, Pal, S, Kvell, K, Sarosi, V, Bai, P, Rue, R, Krymskaya, V, McPhail, D, Porter, A, Pongracz, J E
Format: Article
Language:English
Subjects:
Auranofin
Biosynthesis
Brief Communication
Cell Line, Tumor
Cell lines
Cell Proliferation - physiology
Complications and side effects
Development and progression
Disease Progression
Dosage and administration
Female
Health aspects
Humans
Lung - pathology
Lung Neoplasms - pathology
Lung transplantation
Lymphangioleiomyomatosis - pathology
Medical schools
Mitochondria
Mitochondria - pathology
Mitochondrial diseases
Mitochondrial Diseases - pathology
Organ transplantation
Rapamycin
Rare diseases
Rare Diseases - pathology
Respiratory function
Risk factors
Smooth muscle
Surgery
Therapeutic applications
Young women
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Summary: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.
ISSN:0950-9232
1476-5594
DOI:10.1038/s41388-018-0625-1