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

In vivo delivery is a major barrier to the use of molecular tools for gene modification. Here we demonstrate site-specific gene editing of human cells in vivo in hematopoietic stem cell-engrafted NOD.Cg- Prkdc scid IL2rγ tm1Wjl (abbreviated NOD- scid IL2rγ null ) mice, using biodegradable nanopartic...

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Veröffentlicht in:	Gene therapy 2013-06, Vol.20 (6), p.658-669
Hauptverfasser:	McNeer, N A, Schleifman, E B, Cuthbert, A, Brehm, M, Jackson, A, Cheng, C, Anandalingam, K, Kumar, P, Shultz, L D, Greiner, D L, Mark Saltzman, W, Glazer, P M
Format:	Artikel
Sprache:	eng
Schlagworte:	631/1647/2300 631/1647/350/354 692/700/565/201 Animals Biodegradability Biomedical and Life Sciences Biomedicine Bone marrow CCR2 protein CCR5 protein Cell Biology Cell Line DNA - administration & dosage DNA - chemistry DNA - genetics Gene Expression Gene Targeting Gene Therapy Gene Transfer Techniques Genetic aspects Genetic Therapy Genetically modified organisms Genome editing Green fluorescent protein Health aspects Hematopoietic stem cells Hematopoietic Stem Cells - cytology Human Genetics Humans Intravenous administration Mice Monocyte chemoattractant protein 1 mRNA Nanoparticles Nanoparticles - administration & dosage Nanoparticles - chemistry Nanotechnology original-article Peptide nucleic acids Peptide Nucleic Acids - administration & dosage Peptide Nucleic Acids - chemistry Peptide Nucleic Acids - genetics Physiological aspects Receptors, CCR5 - genetics Rodents Single-stranded DNA Spleen Stem cells
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