A genome editing primer for the hematologist.

No abstract is available for this paper.

• Publication year

  2016

• Venue

  Blood

• Publication date

  2016-05-26

• Fields of study

  Biology, Medicine

• Identifiers
  DOI 10.1182/blood-2016-01-678151PMID 27053532PMCID PMC4882802
• External record

  Open on Semantic Scholar

• Source metadata

  Semantic Scholar, PubMed

• No claims are published for this paper.

• No concepts are published for this paper.

• In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy

  2016cited by this paper
• In vivo gene editing in dystrophic mouse muscle and muscle stem cells

  2016cited by this paper
• Genetic treatment of a molecular disorder: gene therapy approaches to sickle cell disease.

  2016cited by this paper
• Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy

  2016cited by this paper
• Adeno-associated viral vectors for the treatment of hemophilia.

  2016cited by this paper
• 731. High-Fidelity CRISPR-Cas9 Nucleases with No Detectable Genome-Wide Off-Target Effects

  2016cited by this paper
• Homology-driven genome editing in hematopoietic stem and progenitor cells using zinc finger nuclease mRNA and AAV6 donors

  2015cited by this paper
• MLL leukemia induction by genome editing of human CD34+ hematopoietic cells.

  2015cited by this paper
• Efficient modification of CCR5 in primary human hematopoietic cells using a megaTAL nuclease and AAV donor template

  2015cited by this paper
• Intraosseous delivery of lentiviral vectors targeting factor VIII expression in platelets corrects murine hemophilia A.

  2015cited by this paper
• Digenome-seq: genome-wide profiling of CRISPR-Cas9 off-target effects in human cells

  2015cited by this paper
• Highly Specific Epigenome Editing by CRISPR/Cas9 Repressors for Silencing of Distal Regulatory Elements

  2015cited by this paper
• Dynamics of CRISPR-Cas9 genome interrogation in living cells

  2015cited by this paper
• Recurrent AAV2-related insertional mutagenesis in human hepatocellular carcinomas

  2015cited by this paper
• Functional Correction of Large Factor VIII Gene Chromosomal Inversions in Hemophilia A Patient-Derived iPSCs Using CRISPR-Cas9.

  2015cited by this paper
• CRISPR-Cas9 delivery to hard-to-transfect cells via membrane deformation

  2015cited by this paper
• Efficient delivery of nuclease proteins for genome editing in human stem cells and primary cells

  2015cited by this paper
• High-fidelity CRISPR-Cas9 variants with undetectable genome-wide off-targets

  2015cited by this paper
• Cpf1 is a single RNA-guided endonuclease of a class 2 CRISPR-Cas system.

  2015influential reference
• Discovery of cancer drug targets by CRISPR-Cas9 screening of protein domains

  2015cited by this paper
• Transcriptional Heterogeneity and Lineage Commitment in Myeloid Progenitors.

  2015cited by this paper
• Nonviral gene delivery systems by the combination of bubble liposomes and ultrasound.

  2015cited by this paper
• Clinical Scale Zinc Finger Nuclease-mediated Gene Editing of PD-1 in Tumor Infiltrating Lymphocytes for the Treatment of Metastatic Melanoma.

  2015cited by this paper
• CRISPR-Cas9 for medical genetic screens: applications and future perspectives

  2015cited by this paper
• De novo generation of HSCs from somatic and pluripotent stem cell sources.

  2015cited by this paper
• Gene therapy returns to centre stage

  2015cited by this paper
• A mechanism for the suppression of homologous recombination in G1 cells

  2015cited by this paper
• In vivo genome editing using Staphylococcus aureus Cas9

  2015cited by this paper
• BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis

  2015cited by this paper
• Fundamental properties of unperturbed haematopoiesis from stem cells in vivo

  2015cited by this paper
• Self-assembled DNA nanoclews for the efficient delivery of CRISPR-Cas9 for genome editing.

  2015cited by this paper
• Functional annotation of native enhancers with a Cas9 -histone demethylase fusion

  2015cited by this paper
• Broadening the targeting range of Staphylococcus aureus CRISPR-Cas9 by modifying PAM recognition

  2015cited by this paper
• In vivo genome editing of the albumin locus as a platform for protein replacement therapy.

  2015cited by this paper
• Epigenome editing by a CRISPR/Cas9-based acetyltransferase activates genes from promoters and enhancers

  2015cited by this paper
• Clonal hematopoiesis of indeterminate potential and its distinction from myelodysplastic syndromes.

  2015cited by this paper
• Therapeutic genome mutagenesis using synthetic donor DNA and triplex-forming molecules.

  2015cited by this paper
• Efficient intracellular delivery of native proteins.

  2015cited by this paper
• Rationally engineered Cas9 nucleases with improved specificity

  2015cited by this paper
• First Clinical Application of Talen Engineered Universal CAR19 T Cells in B-ALL

  2015cited by this paper
• Resource Engineering Complex Synthetic Transcriptional Programs with CRISPR RNA Scaffolds

  2015cited by this paper
• Creating and evaluating accurate CRISPR-Cas9 scalpels for genomic surgery

  2015cited by this paper
• Engineered CRISPR-Cas9 nucleases with altered PAM specificities

  2015cited by this paper
• Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells.

  2015cited by this paper
• Genome Editing Technologies: Defining a Path to Clinic: Genomic Editing: Establishing Preclinical Toxicology Standards, Bethesda, Maryland 10 June 2014.

  2015cited by this paper
• Somatic mutation in single human neurons tracks developmental and transcriptional history

  2015cited by this paper
• Enabling functional genomics with genome engineering

  2015cited by this paper
• Small molecules enhance CRISPR genome editing in pluripotent stem cells.

  2015cited by this paper
• Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells

  2015cited by this paper
• Multiplex Genome-Edited T-cell Manufacturing Platform for "Off-the-Shelf" Adoptive T-cell Immunotherapies.

  2015cited by this paper
• Functional footprinting of regulatory DNA

  2015cited by this paper
• High-throughput functional genomics using CRISPR–Cas9

  2015cited by this paper
• A CRISPR/Cas9 vector system for tissue-specific gene disruption in zebrafish.

  2015cited by this paper
• Author response: Enhanced homology-directed human genome engineering by controlled timing of CRISPR/Cas9 delivery

  2014cited by this paper
• GUIDE-Seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases

  2014cited by this paper
• Enhanced homology-directed human genome engineering by controlled timing of CRISPR/Cas9 delivery

  2014cited by this paper
• Chimeric antigen receptor T cells for sustained remissions in leukemia.

  2014cited by this paper
• Dissecting the Mechanism of Histone Deacetylase Inhibitors to Enhance the Activity of Zinc Finger Nucleases Delivered by Integrase-Defective Lentiviral Vectors

  2014cited by this paper
• Targeted genomic rearrangements using CRISPR/Cas technology

  2014cited by this paper
• Genome-Scale CRISPR-Cas9 Knockout Screening in Human Cells

  2014cited by this paper
• Development and applications of CRISPR-Cas9 for genome engineering.

  2014cited by this paper
• Efficient ablation of genes in human hematopoietic stem and effector cells using CRISPR/Cas9.

  2014cited by this paper
• Reactivation of developmentally silenced globin genes by forced chromatin looping.

  2014cited by this paper
• Pulmonary Macrophage Transplantation Therapy

  2014cited by this paper
• A guide to genome engineering with programmable nucleases

  2014cited by this paper
• Genome-scale transcriptional activation by an engineered CRISPR-Cas9 complex

  2014cited by this paper
• Targeted Genome Editing in Human Repopulating Hematopoietic Stem Cells

  2014cited by this paper
• Determining the specificities of TALENs, Cas9, and other genome-editing enzymes.

  2014cited by this paper
• Genome-wide detection of DNA double-stranded breaks induced by engineered nucleases

  2014cited by this paper
• Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV.

  2014cited by this paper
• Efficient Delivery of Genome-Editing Proteins In Vitro and In Vivo

  2014cited by this paper
• Efficient genome modification by CRISPR-Cas9 nickase with minimal off-target effects

  2014cited by this paper
• CRISPR-Cas systems for editing, regulating and targeting genomes

  2014cited by this paper
• Genome-Scale CRISPR-Mediated Control of Gene Repression and Activation.

  2014cited by this paper
• Isolation of single-base genome-edited human iPS cells without antibiotic selection

  2014cited by this paper
• Eliminating SCID row: new approaches to SCID.

  2014cited by this paper
• Engineering human tumour-associated chromosomal translocations with the RNA-guided CRISPR–Cas9 system

  2014cited by this paper
• Seamless correction of the sickle cell disease mutation of the HBB gene in human induced pluripotent stem cells using TALENs.

  2014cited by this paper
• Development of gene transfer for induction of antigen-specific tolerance

  2014cited by this paper
• The new frontier of genome engineering with CRISPR-Cas9

  2014cited by this paper
• Clonal dynamics of native haematopoiesis

  2014cited by this paper
• Delivery of Full-Length Factor VIII Using a piggyBac Transposon Vector to Correct a Mouse Model of Hemophilia A

  2014cited by this paper
• Highly-efficient Cas9-mediated transcriptional programming

  2014cited by this paper
• Generation of mouse models of myeloid malignancy with combinatorial genetic lesions using CRISPR-Cas9 genome editing

  2014cited by this paper
• Characterization of Genomic Deletion Efficiency Mediated by Clustered Regularly Interspaced Palindromic Repeats (CRISPR)/Cas9 Nuclease System in Mammalian Cells*♦

  2014cited by this paper
• Gene disruption by cell-penetrating peptide-mediated delivery of Cas9 protein and guide RNA

  2014cited by this paper
• Genome-wide binding of the CRISPR endonuclease Cas9 in mammalian cells

  2014cited by this paper
• Promoterless gene targeting without nucleases ameliorates haemophilia B in mice

  2014cited by this paper
• Intravenous injection of a foamy virus vector to correct canine SCID-X1.

  2014cited by this paper
• Repurposing CRISPR as an RNA-guided platform for sequence-specific control of gene expression.

  2013cited by this paper
• High frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells

  2013cited by this paper
• Chimeric antigen receptor-modified T cells for acute lymphoid leukemia.

  2013cited by this paper
• ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering.

  2013cited by this paper
• Robust ZFN-mediated genome editing in adult hemophilic mice.

  2013cited by this paper
• Locus-specific editing of histone modifications at endogenous enhancers using programmable TALE-LSD1 fusions

  2013cited by this paper
• Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant.

  2013cited by this paper
• megaTALs: a rare-cleaving nuclease architecture for therapeutic genome engineering

  2013cited by this paper
• One-step generation of mice carrying mutations in multiple genes by CRISPR/Cas-mediated genome engineering.

  2013cited by this paper
• RNA-Guided Human Genome Engineering via Cas9

  2013cited by this paper
• Toward eliminating HLA class I expression to generate universal cells from allogeneic donors.

  2013cited by this paper

• CRISPR‐Cas9 delivery strategies and applications: Review and update

  2024influential citation
• Revolutionising healing: Gene Editing's breakthrough against sickle cell disease.

  2024cites this paper
• CRISPR-Cpf1 system and its applications in animal genome editing

  2024cites this paper
• Gene therapy and gene editing strategies in inherited blood disorders.

  2024cites this paper
• Multi-faceted CRISPR/Cas technological innovation aspects in the framework of 3P medicine

  2023cites this paper
• Gene Therapy for Sickle Cell Disease - Moving from the Bench to the Bedside.

  2021cites this paper
• Gene Therapies for Transfusion-Dependent β-Thalassemia

  2021cites this paper
• Acoustofluidic sonoporation for gene delivery to human hematopoietic stem and progenitor cells

  2020cites this paper
• Gene therapies for transfusion dependent β‐thalassemia: Current status and critical criteria for success

  2020cites this paper
• Genome Editing and Hematologic Malignancy.

  2020cites this paper
• Pathophysiology and recent therapeutic insights of sickle cell disease

  2020cites this paper
• Chemistry, manufacturing and controls for gene modified hematopoietic stem cells.

  2019cites this paper
• Editing aberrant splice sites efficiently restores β-globin expression in β-thalassemia.

  2019cites this paper
• Emerging Genetic Therapy for Sickle Cell Disease.

  2019cites this paper
• Genome Editing Technologies: From Bench Side to Bedside

  2018cites this paper
• Engineering chimeric antigen receptor-T cells for cancer treatment

  2018cites this paper
• Genetic therapies for sickle cell disease.

  2018cites this paper
• CRISPR/Cas9 genome engineering in hematopoietic cells.

  2018cites this paper
• CRISP Points on Establishing CRISPR-Cas9 In Vitro Culture Experiments in a Resource Constraint Haematology Oncology Research Lab

  2018cites this paper
• Characterization of Gene Alterations following Editing of the β-Globin Gene Locus in Hematopoietic Stem/Progenitor Cells.

  2017cites this paper
• Homology-Directed Recombination for Enhanced Engineering of Chimeric Antigen Receptor T Cells

  2017cites this paper
• Studying disorders of vertebrate iron and heme metabolism using zebrafish.

  2017cites this paper
• Application of Bioreactor in Stem Cell Culture

  2017cites this paper
• Genetically Modified T-Cell-Based Adoptive Immunotherapy in Hematological Malignancies

  2017cites this paper
• Novel and innovative approaches to the treatment of β-thalassemia

  2017cites this paper
• Therapeutic gene editing in CD34+ hematopoietic progenitors from Fanconi anemia patients

  2017cites this paper
• Jurnal Kedokteran dan Kesehatan Indonesia

  2016cites this paper
• Welcoming a new age for gene therapy in hematology.

  2016cites this paper
• High Efficiency Gene Correction in Hematopoietic Cells by Donor-Template-Free CRISPR/Cas9 Genome Editing

  2016cites this paper