Genetic correction using engineered nucleases for gene therapy applications
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Title
Genetic correction using engineered nucleases for gene therapy applications
Authors
Keywords
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Journal
DEVELOPMENT GROWTH & DIFFERENTIATION
Volume 56, Issue 1, Pages 63-77
Publisher
Wiley
Online
2013-12-12
DOI
10.1111/dgd.12107
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Note: Only part of the references are listed.- One-Step Generation of Mice Carrying Mutations in Multiple Genes by CRISPR/Cas-Mediated Genome Engineering
- (2013) Haoyi Wang et al. CELL
- Double Nicking by RNA-Guided CRISPR Cas9 for Enhanced Genome Editing Specificity
- (2013) F. Ann Ran et al. CELL
- Enhanced Efficiency of Human Pluripotent Stem Cell Genome Editing through Replacing TALENs with CRISPRs
- (2013) Qiurong Ding et al. Cell Stem Cell
- Efficient drug screening and gene correction for treating liver disease using patient-specific stem cells
- (2013) Su Mi Choi et al. HEPATOLOGY
- Efficient Clinical Scale Gene Modification via Zinc Finger Nuclease–Targeted Disruption of the HIV Co-receptor CCR5
- (2013) Dawn A. Maier et al. HUMAN GENE THERAPY
- Gene Correction of a Duchenne Muscular Dystrophy Mutation by Meganuclease-Enhanced Exon Knock-In
- (2013) Linda Popplewell et al. HUMAN GENE THERAPY
- Reading Frame Correction by Targeted Genome Editing Restores Dystrophin Expression in Cells From Duchenne Muscular Dystrophy Patients
- (2013) David G Ousterout et al. MOLECULAR THERAPY
- TALEN-based Gene Correction for Epidermolysis Bullosa
- (2013) Mark J Osborn et al. MOLECULAR THERAPY
- HLA Engineering of Human Pluripotent Stem Cells
- (2013) Laura Riolobos et al. MOLECULAR THERAPY
- Translating dosage compensation to trisomy 21
- (2013) Jun Jiang et al. NATURE
- CAS9 transcriptional activators for target specificity screening and paired nickases for cooperative genome engineering
- (2013) Prashant Mali et al. NATURE BIOTECHNOLOGY
- DNA targeting specificity of RNA-guided Cas9 nucleases
- (2013) Patrick D Hsu et al. NATURE BIOTECHNOLOGY
- High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells
- (2013) Yanfang Fu et al. NATURE BIOTECHNOLOGY
- RNA-guided editing of bacterial genomes using CRISPR-Cas systems
- (2013) Wenyan Jiang et al. NATURE BIOTECHNOLOGY
- Specific elimination of mutant mitochondrial genomes in patient-derived cells by mitoTALENs
- (2013) Sandra R Bacman et al. NATURE MEDICINE
- CRISPR/Cas9 systems targeting β-globin and CCR5 genes have substantial off-target activity
- (2013) Thomas J. Cradick et al. NUCLEIC ACIDS RESEARCH
- Directed evolution of the TALE N-terminal domain for recognition of all 5′ bases
- (2013) Brian M. Lamb et al. NUCLEIC ACIDS RESEARCH
- Efficient genome engineering in human pluripotent stem cells using Cas9 from Neisseria meningitidis
- (2013) Z. Hou et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- RNA-Guided Human Genome Engineering via Cas9
- (2013) P. Mali et al. SCIENCE
- Generating Hypoimmunogenic Human Embryonic Stem Cells by the Disruption of Beta 2-Microglobulin
- (2013) Pengfei Lu et al. Stem Cell Reviews and Reports
- ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering
- (2013) Thomas Gaj et al. TRENDS IN BIOTECHNOLOGY
- Harnessing the CRISPR/Cas9 system to disrupt latent HIV-1 provirus
- (2013) Hirotaka Ebina et al. Scientific Reports
- Development of Multiexon Skipping Antisense Oligonucleotide Therapy for Duchenne Muscular Dystrophy
- (2013) Yoshitsugu Aoki et al. Biomed Research International
- Trisomy Correction in Down Syndrome Induced Pluripotent Stem Cells
- (2012) Li B. Li et al. Cell Stem Cell
- A TALEN Genome-Editing System for Generating Human Stem Cell-Based Disease Models
- (2012) Qiurong Ding et al. Cell Stem Cell
- Gene therapy for Duchenne muscular dystrophy
- (2012) Ingrid E.C. Verhaart et al. CURRENT OPINION IN NEUROLOGY
- Zinc-finger nuclease-mediated gene correction using single AAV vector transduction and enhancement by Food and Drug Administration-approved drugs
- (2012) B L Ellis et al. GENE THERAPY
- Zinc finger nucleases: looking toward translation
- (2012) N J Palpant et al. GENE THERAPY
- Linking disease associations with regulatory information in the human genome
- (2012) M. A. Schaub et al. GENOME RESEARCH
- Genetic Therapeutic Approaches for Duchenne Muscular Dystrophy
- (2012) Helen Foster et al. HUMAN GENE THERAPY
- Unknown
- (2012) Mudit Tyagi et al. MOLECULAR MEDICINE
- Performance comparison of benchtop high-throughput sequencing platforms
- (2012) Nicholas J Loman et al. NATURE BIOTECHNOLOGY
- TALENs: a widely applicable technology for targeted genome editing
- (2012) J. Keith Joung et al. NATURE REVIEWS MOLECULAR CELL BIOLOGY
- Differential integrity of TALE nuclease genes following adenoviral and lentiviral vector gene transfer into human cells
- (2012) Maarten Holkers et al. NUCLEIC ACIDS RESEARCH
- A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity
- (2012) M. Jinek et al. SCIENCE
- Site-specific gene correction of a point mutation in human iPS cells derived from an adult patient with sickle cell disease
- (2011) J. Zou et al. BLOOD
- Generation of Isogenic Pluripotent Stem Cells Differing Exclusively at Two Early Onset Parkinson Point Mutations
- (2011) Frank Soldner et al. CELL
- Targeted chromosomal duplications and inversions in the human genome using zinc finger nucleases
- (2011) H. J. Lee et al. GENOME RESEARCH
- Versatile and Efficient Genome Editing in Human Cells by Combining Zinc-Finger Nucleases With Adeno-Associated Viral Vectors
- (2011) Eva-Maria Händel et al. HUMAN GENE THERAPY
- Endonucleases: tools to correct the dystrophin gene
- (2011) Joel Rousseau et al. JOURNAL OF GENE MEDICINE
- Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study
- (2011) Sebahattin Cirak et al. LANCET
- Efficient and Accurate Homologous Recombination in hESCs and hiPSCs Using Helper-dependent Adenoviral Vectors
- (2011) Emi Aizawa et al. MOLECULAR THERAPY
- Targeted gene correction of α1-antitrypsin deficiency in induced pluripotent stem cells
- (2011) Kosuke Yusa et al. NATURE
- In vivo genome editing restores haemostasis in a mouse model of haemophilia
- (2011) Hojun Li et al. NATURE
- Genetic engineering of human pluripotent cells using TALE nucleases
- (2011) Dirk Hockemeyer et al. NATURE BIOTECHNOLOGY
- An unbiased genome-wide analysis of zinc-finger nuclease specificity
- (2011) Richard Gabriel et al. NATURE BIOTECHNOLOGY
- Revealing off-target cleavage specificities of zinc-finger nucleases by in vitro selection
- (2011) Vikram Pattanayak et al. NATURE METHODS
- Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B
- (2011) Amit C. Nathwani et al. NEW ENGLAND JOURNAL OF MEDICINE
- Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy
- (2011) Nathalie M. Goemans et al. NEW ENGLAND JOURNAL OF MEDICINE
- Extensive protein and DNA backbone sampling improves structure-based specificity prediction for C2H2 zinc fingers
- (2011) Chen Yanover et al. NUCLEIC ACIDS RESEARCH
- Genetic correction and analysis of induced pluripotent stem cells from a patient with gyrate atrophy
- (2011) S. E. Howden et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- In Situ Genetic Correction of the Sickle Cell Anemia Mutation in Human Induced Pluripotent Stem Cells Using Engineered Zinc Finger Nucleases
- (2011) Vittorio Sebastiano et al. STEM CELLS
- Modeling Disease in Human ESCs Using an Efficient BAC-Based Homologous Recombination System
- (2010) Hoseok Song et al. Cell Stem Cell
- Meganucleases can restore the reading frame of a mutated dystrophin
- (2010) P Chapdelaine et al. GENE THERAPY
- Frequent Endonuclease Cleavage at Off-target Locations In Vivo
- (2010) Lisa M Petek et al. MOLECULAR THERAPY
- Engineering of Human Pluripotent Stem Cells by AAV-mediated Gene Targeting
- (2010) Iram F Khan et al. MOLECULAR THERAPY
- Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia
- (2010) Marina Cavazzana-Calvo et al. NATURE
- 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
- Efficacy of Gene Therapy for X-Linked Severe Combined Immunodeficiency
- (2010) Salima Hacein-Bey-Abina et al. NEW ENGLAND JOURNAL OF MEDICINE
- The I-CreI meganuclease and its engineered derivatives: applications from cell modification to gene therapy
- (2010) S. Arnould et al. PROTEIN ENGINEERING DESIGN & SELECTION
- Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases
- (2009) Dirk Hockemeyer et al. NATURE BIOTECHNOLOGY
- Mechanisms of change in gene copy number
- (2009) P. J. Hastings et al. NATURE REVIEWS GENETICS
- Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency
- (2009) Alessandro Aiuti et al. NEW ENGLAND JOURNAL OF MEDICINE
- Retroviral vector silencing during iPS cell induction: An epigenetic beacon that signals distinct pluripotent states
- (2008) Akitsu Hotta et al. JOURNAL OF CELLULAR BIOCHEMISTRY
- Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases
- (2008) Elena E Perez et al. NATURE BIOTECHNOLOGY
- Targeted gene inactivation in zebrafish using engineered zinc-finger nucleases
- (2008) Xiangdong Meng et al. NATURE BIOTECHNOLOGY
- Highly efficient transient gene expression and gene targeting in primate embryonic stem cells with helper-dependent adenoviral vectors
- (2008) K. Suzuki et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Robust, Persistent Transgene Expression in Human Embryonic Stem Cells Is Achieved with AAVS1-Targeted Integration
- (2007) Joseph R. Smith et al. STEM CELLS
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