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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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書誌詳細
主要な著者: Zhang, Pei, Huang, Angela, Morales-Ruiz, Manuel, Starcher, Barry C., Huang, Yan, Sessa, William C., Niklason, Laura E., Giordano, Frank J.
フォーマット: Artigo
言語:Inglês
出版事項: Mary Ann Liebert, Inc. 2012
主題:
オンライン・アクセス: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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