Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors
Published 2015 View Full Article
- Home
- Publications
- Publication Search
- Publication Details
Title
Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors
Authors
Keywords
-
Journal
NATURE BIOTECHNOLOGY
Volume 33, Issue 12, Pages 1256-1263
Publisher
Springer Nature
Online
2015-11-10
DOI
10.1038/nbt.3408
References
Ask authors/readers for more resources
Related references
Note: Only part of the references are listed.- Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells
- (2015) M. D. Hoban et al. BLOOD
- Improved specificity of TALE-based genome editing using an expanded RVD repertoire
- (2015) Jeffrey C Miller et al. NATURE METHODS
- A platform for rapid generation of single and multiplexed reporters in human iPSC lines
- (2015) Ying Pei et al. Scientific Reports
- Adeno-associated virus inverted terminal repeats stimulate gene editing
- (2014) M L Hirsch GENE THERAPY
- Targeted genome editing in human repopulating haematopoietic stem cells
- (2014) Pietro Genovese et al. NATURE
- Promoterless gene targeting without nucleases ameliorates haemophilia B in mice
- (2014) A. Barzel et al. NATURE
- Broad specificity profiling of TALENs results in engineered nucleases with improved DNA-cleavage specificity
- (2014) John P Guilinger et al. NATURE METHODS
- A guide to genome engineering with programmable nucleases
- (2014) Hyongbum Kim et al. NATURE REVIEWS GENETICS
- Gene Editing of CCR5 in Autologous CD4 T Cells of Persons Infected with HIV
- (2014) Pablo Tebas et al. NEW ENGLAND JOURNAL OF MEDICINE
- Gene Therapy for Wiskott-Aldrich Syndrome--Long-Term Efficacy and Genotoxicity
- (2014) C. J. Braun et al. Science Translational Medicine
- Engraftment and Lineage Potential of Adult Hematopoietic Stem and Progenitor Cells Is Compromised Following Short-Term Culture in the Presence of an Aryl Hydrocarbon Receptor Antagonist
- (2014) Angel Gu et al. Human Gene Therapy Methods
- Double Nicking by RNA-Guided CRISPR Cas9 for Enhanced Genome Editing Specificity
- (2013) F. Ann Ran et al. CELL
- Optimizing the transduction efficiency of capsid-modified AAV6 serotype vectors in primary human hematopoietic stem cells in vitro and in a xenograft mouse model in vivo
- (2013) Liujiang Song et al. CYTOTHERAPY
- Mechanisms of Retroviral Integration and Mutagenesis
- (2013) Alessia Cavazza et al. HUMAN GENE THERAPY
- Pre-clinical Modeling of CCR5 Knockout in Human Hematopoietic Stem Cells by Zinc Finger Nucleases Using Humanized Mice
- (2013) U. Hofer et al. JOURNAL OF INFECTIOUS DISEASES
- Targeted Gene Addition in Human Epithelial Stem Cells by Zinc-finger Nuclease-mediated Homologous Recombination
- (2013) Andrea Coluccio et al. MOLECULAR THERAPY
- Genomic Editing of the HIV-1 Coreceptor CCR5 in Adult Hematopoietic Stem and Progenitor Cells Using Zinc Finger Nucleases
- (2013) Lijing Li et al. MOLECULAR THERAPY
- High-Efficiency Transduction of Primary Human Hematopoietic Stem Cells and Erythroid Lineage-Restricted Expression by Optimized AAV6 Serotype Vectors In Vitro and in a Murine Xenograft Model In Vivo
- (2013) Liujiang Song et al. PLoS One
- Corepressor-dependent silencing of fetal hemoglobin expression by BCL11A
- (2013) J. Xu et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome
- (2013) A. Aiuti et al. SCIENCE
- Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy
- (2013) A. Biffi et al. SCIENCE
- ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering
- (2013) Thomas Gaj et al. TRENDS IN BIOTECHNOLOGY
- Hematopoiesis: A Human Perspective
- (2012) Sergei Doulatov et al. Cell Stem Cell
- Targeted gene addition to a predetermined site in the human genome using a ZFN-based nicking enzyme
- (2012) J. Wang et al. GENOME RESEARCH
- Fast gapped-read alignment with Bowtie 2
- (2012) Ben Langmead et al. NATURE METHODS
- An unbiased genome-wide analysis of zinc-finger nuclease specificity
- (2011) Richard Gabriel et al. NATURE BIOTECHNOLOGY
- Site-specific integration and tailoring of cassette design for sustainable gene transfer
- (2011) Angelo Lombardo et al. NATURE METHODS
- Chemically defined conditions for human iPSC derivation and culture
- (2011) Guokai Chen et al. NATURE METHODS
- Isolation of Single Human Hematopoietic Stem Cells Capable of Long-Term Multilineage Engraftment
- (2011) F. Notta et al. SCIENCE
- Functional genomics, proteomics, and regulatory DNA analysis in isogenic settings using zinc finger nuclease-driven transgenesis into a safe harbor locus in the human genome
- (2010) R. C. DeKelver et al. GENOME RESEARCH
- Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo
- (2010) Nathalia Holt et al. NATURE BIOTECHNOLOGY
- Enhancing zinc-finger-nuclease activity with improved obligate heterodimeric architectures
- (2010) Yannick Doyon et al. NATURE METHODS
- Genome editing with engineered zinc finger nucleases
- (2010) Fyodor D. Urnov et al. NATURE REVIEWS GENETICS
- Gene targeting by homologous recombination in mouse zygotes mediated by zinc-finger nucleases
- (2010) M. Meyer et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Pseudotyped recombinant adeno-associated viral vectors mediate efficient gene transfer into primary human CD34+ peripheral blood progenitor cells
- (2009) Marlon R. Veldwijk et al. CYTOTHERAPY
- Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy
- (2009) N. Cartier et al. SCIENCE
- Production of CFTR-null and CFTR-ΔF508 heterozygous pigs by adeno-associated virus–mediated gene targeting and somatic cell nuclear transfer
- (2008) Christopher S. Rogers et al. JOURNAL OF CLINICAL INVESTIGATION
- Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1
- (2008) Salima Hacein-Bey-Abina et al. JOURNAL OF CLINICAL INVESTIGATION
- Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases
- (2008) Elena E Perez et al. NATURE BIOTECHNOLOGY
- Regulation of DNA repair throughout the cell cycle
- (2008) Dana Branzei et al. NATURE REVIEWS MOLECULAR CELL BIOLOGY
- Robust, Persistent Transgene Expression in Human Embryonic Stem Cells Is Achieved with AAVS1-Targeted Integration
- (2007) Joseph R. Smith et al. STEM CELLS
Find Funding. Review Successful Grants.
Explore over 25,000 new funding opportunities and over 6,000,000 successful grants.
ExploreBecome a Peeref-certified reviewer
The Peeref Institute provides free reviewer training that teaches the core competencies of the academic peer review process.
Get Started