Gene therapy restores adipose tissue and metabolic health in a pre-clinical mouse model of lipodystrophy

Congenital generalized lipodystrophy type 2 is a serious multisystem disorder with limited treatment options. It is caused by mutations affecting the BSCL2 gene, which encodes the protein seipin. Patients with congenital generalized lipodystrophy type 2 lack both metabolic and mechanical adipose tis...

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Bibliographic Details
Main Authors: Nadine Sommer, Ahlima Roumane, Weiping Han, Mirela Delibegović, Justin J. Rochford, George D. Mcilroy
Format: Article
Language:English
Published: Elsevier 2022-12-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050122001395