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

E. M. M. Abdelwahab, S. Pal, K. Kvell, V. Sarosi, P. Bai, R. Rue, V. Krymskaya, D. McPhail, A. Porter, J. E. Pongracz (Corresponding Author)

Research output: Contribution to journalArticle

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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.
Original languageEnglish
Pages (from-to)3093-3101
Number of pages9
JournalOncogene
Volume38
Early online date20 Dec 2018
DOIs
Publication statusPublished - 2019

Fingerprint

Lymphangioleiomyomatosis
Rare Diseases
Lung
Lung Transplantation
Organelle Biogenesis
Pneumothorax
Smooth Muscle
Disease Progression
Therapeutics
Cell Line
Growth

Keywords

  • BIOGENESIS
  • CELL
  • CHAIN
  • CYTOCHROME-C
  • ESTROGEN
  • FEATURES
  • GENE
  • PHOSPHORYLATION
  • TSC2

ASJC Scopus subject areas

  • Genetics
  • Molecular Biology
  • Cancer Research

Cite this

Mitochondrial dysfunction is a key determinant of the rare disease lymphangioleiomyomatosis and provides a novel therapeutic target. / Abdelwahab, E. M. M.; Pal, S.; Kvell, K.; Sarosi, V.; Bai, P.; Rue, R.; Krymskaya, V.; McPhail, D.; Porter, A.; Pongracz, J. E. (Corresponding Author).

In: Oncogene, Vol. 38, 2019, p. 3093-3101.

Research output: Contribution to journalArticle

Abdelwahab, EMM, Pal, S, Kvell, K, Sarosi, V, Bai, P, Rue, R, Krymskaya, V, McPhail, D, Porter, A & Pongracz, JE 2019, 'Mitochondrial dysfunction is a key determinant of the rare disease lymphangioleiomyomatosis and provides a novel therapeutic target', Oncogene, vol. 38, pp. 3093-3101. https://doi.org/10.1038/s41388-018-0625-1
Abdelwahab, E. M. M. ; Pal, S. ; Kvell, K. ; Sarosi, V. ; Bai, P. ; Rue, R. ; Krymskaya, V. ; McPhail, D. ; Porter, A. ; Pongracz, J. E. / Mitochondrial dysfunction is a key determinant of the rare disease lymphangioleiomyomatosis and provides a novel therapeutic target. In: Oncogene. 2019 ; Vol. 38. pp. 3093-3101.
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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.",
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