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Verfasst von:Kallenbach, Klaus [VerfasserIn]   i
 Remes, Anca [VerfasserIn]   i
 Müller, Oliver J. [VerfasserIn]   i
 Arif, Rawa [VerfasserIn]   i
 Zaradzki, Marcin [VerfasserIn]   i
 Wagner, Andreas H. [VerfasserIn]   i
Titel:Translational medicine
Titelzusatz:towards gene therapy of marfan syndrome
Verf.angabe:Klaus Kallenbach, Anca Remes, Oliver J. Müller, Rawa Arif, Marcin Zaradzki and Andreas H. Wagner
E-Jahr:2022
Jahr:6 July 2022
Umfang:11 S.
Fussnoten:Gesehen am 05.09.2022
Titel Quelle:Enthalten in: Journal of Clinical Medicine
Ort Quelle:Basel : MDPI, 2012
Jahr Quelle:2022
Band/Heft Quelle:11(2022), 14, Artikel-ID 3934, Seite 1-11
ISSN Quelle:2077-0383
Abstract: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.
DOI:doi:10.3390/jcm11143934
URL:kostenfrei: Volltext: https://doi.org/10.3390/jcm11143934
 kostenfrei: Volltext: https://www.mdpi.com/2077-0383/11/14/3934
 DOI: https://doi.org/10.3390/jcm11143934
Datenträger:Online-Ressource
Sprache:eng
Sach-SW:aorta
 aortic surgery
 gene therapy
 marfan syndrome
 TGF-β
 translational therapy
K10plus-PPN:1815779896
Verknüpfungen:→ Zeitschrift
 
 
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