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Translational medicine: towards gene therapy of marfan syndrome

Marfan syndrome (MFS) is one of the most common inherited disorders of connective tissue caused by mutations of the fibrillin-1 gene (FBN1). Vascular abnormalities, such as the enlargement of the aorta with the risk of life-threatening rupture are frequently observed. However, current treatment is l...

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Bibliographic Details
Main Authors: Kallenbach, Klaus (Author) , Remes, Anca (Author) , Müller, Oliver J. (Author) , Arif, Rawa (Author) , Zaradzki, Marcin (Author) , Wagner, Andreas H. (Author)
Format: Article (Journal)
Language:English
Published: 6 July 2022
In: Journal of Clinical Medicine
Year: 2022, Volume: 11, Issue: 14, Pages: 1-11
ISSN:2077-0383
DOI:10.3390/jcm11143934
Online Access:Verlag, kostenfrei, Volltext: https://doi.org/10.3390/jcm11143934
Verlag, kostenfrei, Volltext: https://www.mdpi.com/2077-0383/11/14/3934
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Author Notes:Klaus Kallenbach, Anca Remes, Oliver J. Müller, Rawa Arif, Marcin Zaradzki and Andreas H. Wagner
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Summary:Marfan syndrome (MFS) is one of the most common inherited disorders of connective tissue caused by mutations of the fibrillin-1 gene (FBN1). Vascular abnormalities, such as the enlargement of the aorta with the risk of life-threatening rupture are frequently observed. However, current treatment is limited and therapeutic options focus solely on symptomatic therapy. Gene therapy focuses on genetically modifying cells to produce a therapeutic effect and may be a promising treatment option for MFS. Here, we first provide an overview of the historical background and characterization of MFS. Subsequently, we summarise current gene therapy options and possible translational concepts for this inherited disorder that affects connective tissue.
Item Description:Gesehen am 05.09.2022
Physical Description:Online Resource
ISSN:2077-0383
DOI:10.3390/jcm11143934

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