Systemic delivery of triplex-forming PNA and donor DNA by nanoparticles mediates site-specific genome editing of human hematopoietic cells in vivo

Gene Therapy

Volumn 20, Issue 6, 2013, Pages 658-669

  McNeer, N A ^a   Schleifman, E B ^a   Cuthbert, A ^b   Brehm, M ^b   Jackson, A ^c   Cheng, C ^a   Anandalingam, K ^a   Kumar, P ^c   Shultz, L D ^d   Greiner, D L ^b   Mark Saltzman, W ^a   Glazer, P M ^a  

^a YALE UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b University of Massachusetts Worcester   (United States)

^c YALE UNIVERSITY   (United States)

^d JACKSON LABORATORY   (United States)

Author keywords

CCR5; Gene delivery; Gene modification; Nanoparticles; Peptide nucleic acid; Triple forming oligonucleotide

Indexed keywords

ENHANCED GREEN FLUORESCENT PROTEIN; HEMOGLOBIN BETA CHAIN; MESSENGER RNA; NANOPARTICLE;

ANIMAL EXPERIMENT; ANIMAL TISSUE; AQUEOUS SOLUTION; ARTICLE; BIODEGRADATION; CELL DIFFERENTIATION; CELL SURVIVAL; CONTROLLED STUDY; DNA SEQUENCE; HEMATOPOIETIC CELL; HUMAN; IN VITRO STUDY; IN VIVO GENE TRANSFER; IN VIVO STUDY; MOLECULAR WEIGHT; MOUSE; NONHUMAN; PH; PRIORITY JOURNAL; PROTEIN EXPRESSION; STOP CODON;

ANIMALS; CELL LINE; DNA; GENE TARGETING; GENE TRANSFER TECHNIQUES; GENETIC THERAPY; HEMATOPOIETIC STEM CELLS; HUMANS; MICE; NANOPARTICLES; PEPTIDE NUCLEIC ACIDS; RECEPTORS, CCR5;

MUS;

EID: 84882283204     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2012.82     Document Type: Article

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