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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

Authors
Journal
Human Gene Therapy
1043-0342
Publisher
Mary Ann Liebert
Publication Date
Volume
23
Issue
11
Identifiers
DOI: 10.1089/hum.2011.201
Keywords
  • Research Articles
Disciplines
  • Biology
  • Engineering

Abstract

Zhang and colleagues evaluate the feasibility of using engineered zinc finger protein transcription factors (ZFPs) to induce elastin expression from the remaining wild-type allele in the context of Williams-Beuren syndrome and supravalvular aortic stenosis elastin haploinsufficiency syndrome. ZFP induced expression of the major elastin variants in their natural stoichiometric pattern, without inducing expression from the mutant allele.

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