In vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle delivery
Published 2016 View Full Article
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Title
In vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle delivery
Authors
Keywords
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Journal
Nature Communications
Volume 7, Issue -, Pages 13304
Publisher
Springer Nature
Online
2016-10-26
DOI
10.1038/ncomms13304
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Note: Only part of the references are listed.- Improved Hematopoietic Differentiation Efficiency of Gene-Corrected Beta-Thalassemia Induced Pluripotent Stem Cells by CRISPR/Cas9 System
- (2015) Bing Song et al. STEM CELLS AND DEVELOPMENT
- Nanoparticles that deliver triplex-forming peptide nucleic acid molecules correct F508del CFTR in airway epithelium
- (2015) Nicole Ali McNeer et al. Nature Communications
- Both TALENs and CRISPR/Cas9 directly target the HBB IVS2–654 (C > T) mutation in β-thalassemia-derived iPSCs
- (2015) Peng Xu et al. Scientific Reports
- Single-Stranded γPNAs for In Vivo Site-Specific Genome Editing via Watson-Crick Recognition
- (2014) Raman Bahal et al. CURRENT GENE THERAPY
- Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype
- (2014) Hao Yin et al. NATURE BIOTECHNOLOGY
- The new frontier of genome engineering with CRISPR-Cas9
- (2014) J. A. Doudna et al. SCIENCE
- Modified Poly(lactic-co-glycolic Acid) Nanoparticles for Enhanced Cellular Uptake and Gene Editing in the Lung
- (2014) Rachel J. Fields et al. Advanced Healthcare Materials
- CRISPR/Cas9 systems targeting β-globin and CCR5 genes have substantial off-target activity
- (2013) Thomas J. Cradick et al. NUCLEIC ACIDS RESEARCH
- Site-specific Genome Editing in PBMCs With PLGA Nanoparticle-delivered PNAs Confers HIV-1 Resistance in Humanized Mice
- (2013) Erica B Schleifman et al. Molecular Therapy-Nucleic Acids
- Systemic delivery of triplex-forming PNA and donor DNA by nanoparticles mediates site-specific genome editing of human hematopoietic cells in vivo
- (2012) N A McNeer et al. GENE THERAPY
- Sequence-Unrestricted, Watson-Crick Recognition of Double Helical B-DNA by (R)-MiniPEG-γPNAs
- (2011) Raman Bahal et al. CHEMBIOCHEM
- Targeted Disruption of the CCR5 Gene in Human Hematopoietic Stem Cells Stimulated by Peptide Nucleic Acids
- (2011) Erica B. Schleifman et al. CHEMISTRY & BIOLOGY
- Zinc-Finger Nuclease Based Genome Surgery: Its All About Specificity
- (2011) Eva-Maria Handel et al. CURRENT GENE THERAPY
- Synthesis and Characterization of Conformationally Preorganized, (R)-Diethylene Glycol-Containing γ-Peptide Nucleic Acids with Superior Hybridization Properties and Water Solubility
- (2011) Bichismita Sahu et al. JOURNAL OF ORGANIC CHEMISTRY
- Nanoparticles Deliver Triplex-forming PNAs for Site-specific Genomic Recombination in CD34+ Human Hematopoietic Progenitors
- (2010) Nicole A McNeer et al. MOLECULAR THERAPY
- RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice
- (2009) S. Svasti et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Repair of DNA lesions associated with triplex-forming oligonucleotides
- (2008) Joanna Y. Chin et al. MOLECULAR CARCINOGENESIS
- In vivo selection of genetically modified erythroblastic progenitors leads to long-term correction of -thalassemia
- (2008) A. Miccio et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
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