Targeted gene addition in human CD34+ hematopoietic cells for correction of X-linked chronic granulomatous disease
Published 2016 View Full Article
- Home
- Publications
- Publication Search
- Publication Details
Title
Targeted gene addition in human CD34+ hematopoietic cells for correction of X-linked chronic granulomatous disease
Authors
Keywords
-
Journal
NATURE BIOTECHNOLOGY
Volume 34, Issue 4, Pages 424-429
Publisher
Springer Nature
Online
2016-03-08
DOI
10.1038/nbt.3513
References
Ask authors/readers for more resources
Related references
Note: Only part of the references are listed.- Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors
- (2015) Jianbin Wang et al. NATURE BIOTECHNOLOGY
- Functional footprinting of regulatory DNA
- (2015) Jeff Vierstra et al. NATURE METHODS
- Genome Engineering with Targetable Nucleases
- (2014) Dana Carroll Annual Review of Biochemistry
- Common Severe Infections in Chronic Granulomatous Disease
- (2014) B. E. Marciano et al. CLINICAL INFECTIOUS DISEASES
- Genome engineering with TALENs and ZFNs: Repair pathways and donor design
- (2014) Dana Carroll et al. METHODS
- Targeted genome editing in human repopulating haematopoietic stem cells
- (2014) Pietro Genovese et al. NATURE
- Gene Editing of CCR5 in Autologous CD4 T Cells of Persons Infected with HIV
- (2014) Pablo Tebas et al. NEW ENGLAND JOURNAL OF MEDICINE
- 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
- 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
- Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy
- (2013) A. Biffi et al. SCIENCE
- Ubiquitous high-level gene expression in hematopoietic lineages provides effective lentiviral gene therapy of murine Wiskott-Aldrich syndrome
- (2012) A. Astrakhan et al. BLOOD
- 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
- 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
- Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease
- (2010) Stefan Stein et al. NATURE MEDICINE
- Residual NADPH Oxidase and Survival in Chronic Granulomatous Disease
- (2010) Douglas B. Kuhns et al. NEW ENGLAND JOURNAL OF MEDICINE
- Efficacy of Gene Therapy for X-Linked Severe Combined Immunodeficiency
- (2010) Salima Hacein-Bey-Abina et al. NEW ENGLAND JOURNAL OF MEDICINE
- Zinc-finger nuclease-driven targeted integration into mammalian genomes using donors with limited chromosomal homology
- (2010) Salvatore J. Orlando et al. NUCLEIC ACIDS RESEARCH
Find Funding. Review Successful Grants.
Explore over 25,000 new funding opportunities and over 6,000,000 successful grants.
ExploreAsk a Question. Answer a Question.
Quickly pose questions to the entire community. Debate answers and get clarity on the most important issues facing researchers.
Get Started