Preclinical evaluation of candidate “kill or cure” strategies to treat MFN2-related lipodystrophy

Preclinical evaluation of candidate “kill or cure” strategies to treat MFN2-related lipodystrophy

Abstract Background The mitofusin 2 (MFN2) R707W mutation causes debilitating human lipodystrophy featuring lower body adipose loss, upper body adipose hyperplasia, and dyslipidaemic insulin resistance. Mechanical complications include airway compromise due to head and neck adipose overgrowth. This...

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Main Authors: Ineke Luijten, Xiong Weng, Ula Kibildyte, Jana Buchan, Ami Onishi, Jake Mann, Eleanor McKay, David Savage, Robert K. Semple
Format: Article
Language:English
Published: BMC 2025-08-01
Series:Molecular Medicine
Subjects:
Mitofusin
MFN2
Lipodystrophy
Multiple symmetrical lipomatosis
Alcohol
Rapamycin
Online Access:https://doi.org/10.1186/s10020-025-01314-2
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https://doi.org/10.1186/s10020-025-01314-2

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