Engineered Zinc-Finger Proteins Can Compensate Genetic Haploinsufficiency by Transcriptional Activation of the Wild-Type Allele: Application to Willams-Beuren Syndrome and Supravalvular Aortic Stenosis

Williams-Beuren syndrome (WBS) and supravalvular aortic stenosis (SVAS) are genetic syndromes marked by the propensity to develop severe vascular stenoses. Vascular lesions in both syndromes are caused by haploinsufficiency of the elastin gene. We used these distinct genetic syndromes as models to e...

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Bibliographische Detailangaben
Hauptverfasser: Zhang, Pei, Huang, Angela, Morales-Ruiz, Manuel, Starcher, Barry C., Huang, Yan, Sessa, William C., Niklason, Laura E., Giordano, Frank J.
Format: Artigo
Sprache:Inglês
Veröffentlicht: Mary Ann Liebert, Inc. 2012
Schlagworte:
Research Articles
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3498887/
https://ncbi.nlm.nih.gov/pubmed/22891920
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2011.201
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