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

Human Gene Therapy

Volumn 23, Issue 11, 2012, Pages 1186-1199

  Zhang, Pei ^a   Huang, Angela ^a   Morales Ruiz, Manuel ^b   Starcher, Barry C ^c   Huang, Yan ^a   Sessa, William C ^a   Niklason, Laura E ^a   Giordano, Frank J ^a  

^a YALE UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b INSTITUT D INVESTIGACIONS BIOMÈDIQUES AUGUST PI I SUNYER IDIBAPS   (Spain)

^c UNIVERSITY OF TEXAS HEALTH CENTER   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ELASTIN; MUTANT PROTEIN; ZINC FINGER PROTEIN;

ADULT; ALLELE; AORTA SUPRAVALVULAR STENOSIS; ARTICLE; BIOGENESIS; BLOOD VESSEL; CONTROLLED STUDY; DRUG MECHANISM; ELASTIN SYNTHESIS; EMBRYO; GENE EXPRESSION; GENE MUTATION; GENE REPLACEMENT THERAPY; GENE TARGETING; GENETIC ENGINEERING; GENETIC TRANSFECTION; GENETIC VARIABILITY; HAPLOINSUFFICIENCY; HUMAN; HUMAN CELL; NONSENSE MEDIATED MRNA DECAY; STOICHIOMETRY; TISSUE ENGINEERING; TRANSCRIPTION INITIATION; WILLIAMS BEUREN SYNDROME;

ALLELES; AORTIC STENOSIS, SUPRAVALVULAR; CELL LINE; CELL MOVEMENT; CELL PROLIFERATION; DOSAGE COMPENSATION, GENETIC; ELASTIN; GENE EXPRESSION; GENE EXPRESSION REGULATION; HAPLOINSUFFICIENCY; HUMANS; MUTATION; NONSENSE MEDIATED MRNA DECAY; ORGAN SPECIFICITY; PROTEIN ENGINEERING; TRANSCRIPTIONAL ACTIVATION; WILLIAMS SYNDROME; ZINC FINGERS;

EID: 84869003173     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2011.201     Document Type: Article

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