Published in Nat Med on February 01, 2015
Safety Study of CALAA-01 to Treat Solid Tumor Cancers | NCT00689065
In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy. Science (2015) 4.18
In vivo genome editing via CRISPR/Cas9 mediated homology-independent targeted integration. Nature (2016) 2.37
Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage. Nature (2016) 2.15
Gene therapy returns to centre stage. Nature (2015) 1.95
High-Throughput, High-Resolution Mapping of Protein Localization in Mammalian Brain by In Vivo Genome Editing. Cell (2016) 1.53
Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo. Nat Biotechnol (2016) 1.44
CD7-edited T cells expressing a CD7-specific CAR for the therapy of T-cell malignancies. Blood (2017) 1.41
Pluripotent stem cells in disease modelling and drug discovery. Nat Rev Mol Cell Biol (2016) 1.24
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Adenovirus-Mediated Somatic Genome Editing of Pten by CRISPR/Cas9 in Mouse Liver in Spite of Cas9-Specific Immune Responses. Hum Gene Ther (2015) 1.18
Applications of CRISPR-Cas systems in neuroscience. Nat Rev Neurosci (2015) 1.18
CRISPR/Cas9 cleavage of viral DNA efficiently suppresses hepatitis B virus. Sci Rep (2015) 1.14
RS-1 enhances CRISPR/Cas9- and TALEN-mediated knock-in efficiency. Nat Commun (2016) 1.10
Gene therapies that restore dystrophin expression for the treatment of Duchenne muscular dystrophy. Hum Genet (2016) 1.04
Error-Prone Repair of DNA Double-Strand Breaks. J Cell Physiol (2016) 1.04
Prospects for gene-engineered T cell immunotherapy for solid cancers. Nat Med (2016) 1.03
Human induced pluripotent stem cell-derived cardiomyocytes: insights into molecular, cellular, and functional phenotypes. Circ Res (2015) 1.02
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Enabling functional genomics with genome engineering. Genome Res (2015) 0.90
Knock-in of large reporter genes in human cells via CRISPR/Cas9-induced homology-dependent and independent DNA repair. Nucleic Acids Res (2016) 0.89
Precision cancer mouse models through genome editing with CRISPR-Cas9. Genome Med (2015) 0.89
Cornerstones of CRISPR-Cas in drug discovery and therapy. Nat Rev Drug Discov (2016) 0.88
New and emerging targeted therapies for cystic fibrosis. BMJ (2016) 0.87
The New State of the Art: Cas9 for Gene Activation and Repression. Mol Cell Biol (2015) 0.87
Digital detection of endonuclease mediated gene disruption in the HIV provirus. Sci Rep (2016) 0.86
Genome-editing technologies for gene correction of hemophilia. Hum Genet (2016) 0.85
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Synthetic biology in cell-based cancer immunotherapy. Trends Biotechnol (2015) 0.84
Multiplex genome editing to generate universal CAR T cells resistant to PD1 inhibition. Clin Cancer Res (2016) 0.82
Companion animals: Translational scientist's new best friends. Sci Transl Med (2015) 0.82
CRISPR-Cas9 Targeting of PCSK9 in Human Hepatocytes In Vivo-Brief Report. Arterioscler Thromb Vasc Biol (2016) 0.82
Use of genome-editing tools to treat sickle cell disease. Hum Genet (2016) 0.82
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Advances in targeted and immunobased therapies for colorectal cancer in the genomic era. Onco Targets Ther (2016) 0.81
A non-inheritable maternal Cas9-based multiple-gene editing system in mice. Sci Rep (2016) 0.81
Future of breeding by genome editing is in the hands of regulators. GM Crops Food (2015) 0.81
Cystic Fibrosis Gene Therapy in the UK and Elsewhere. Hum Gene Ther (2015) 0.80
Progress and Prospects of Anti-HBV Gene Therapy Development. Int J Mol Sci (2015) 0.80
Biotechnological strategies and tools for Plum pox virus resistance: trans-, intra-, cis-genesis, and beyond. Front Plant Sci (2015) 0.80
Emerging therapies for mitochondrial disorders. Brain (2016) 0.80
Induced Pluripotency and Gene Editing in Disease Modelling: Perspectives and Challenges. Int J Mol Sci (2015) 0.79
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Harnessing human ADAR2 for RNA repair - Recoding a PINK1 mutation rescues mitophagy. Nucleic Acids Res (2016) 0.78
Optimization of a multiplex CRISPR/Cas system for use as an antiviral therapeutic. Methods (2015) 0.78
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Patient-derived induced pluripotent stem cells in cancer research and precision oncology. Nat Med (2016) 0.77
High content analysis platform for optimization of lipid mediated CRISPR-Cas9 delivery strategies in human cells. Acta Biomater (2015) 0.77
New directions for the treatment of adrenal insufficiency. Front Endocrinol (Lausanne) (2015) 0.76
Engineering prokaryotic channels for control of mammalian tissue excitability. Nat Commun (2016) 0.76
The complexity of epigenetic diseases. J Pathol (2015) 0.76
Gene Editing for the Efficient Correction of a Recurrent COL7A1 Mutation in Recessive Dystrophic Epidermolysis Bullosa Keratinocytes. Mol Ther Nucleic Acids (2016) 0.76
Utilizing cell-based therapeutics to overcome immune evasion in hematologic malignancies. Blood (2016) 0.76
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Applications of CRISPR/Cas9 for Gene Editing in Hereditary Movement Disorders. J Mov Disord (2016) 0.75
Development of autologous blood cell therapies. Exp Hematol (2016) 0.75
In Vivo Delivery Systems for Therapeutic Genome Editing. Int J Mol Sci (2016) 0.75
Clinical Applications of Genome Editing to HIV Cure. AIDS Patient Care STDS (2016) 0.75
RNA-guided transcriptional activation via CRISPR/dCas9 mimics overexpression phenotypes in Arabidopsis. PLoS One (2017) 0.75
Using intron splicing trick for preferential gene expression in transduced cells: an approach for suicide gene therapy. Cancer Gene Ther (2015) 0.75
May I Cut in? Gene Editing Approaches in Human Induced Pluripotent Stem Cells. Cells (2017) 0.75
GUIDEseq: a bioconductor package to analyze GUIDE-Seq datasets for CRISPR-Cas nucleases. BMC Genomics (2017) 0.75
In vivo versus ex vivo CRISPR therapies for retinal dystrophy. Expert Rev Ophthalmol (2016) 0.75
CRISPR-Cas9: a promising tool for gene editing on induced pluripotent stem cells. Korean J Intern Med (2017) 0.75
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CRISPR/Cas9-AAV Mediated Knock-in at NRL Locus in Human Embryonic Stem Cells. Mol Ther Nucleic Acids (2016) 0.75
Editing VEGFR2 Blocks VEGF-Induced Activation of Akt and Tube Formation. Invest Ophthalmol Vis Sci (2017) 0.75
Sendai virus, an RNA virus with no risk of genomic integration, delivers CRISPR/Cas9 for efficient gene editing. Mol Ther Methods Clin Dev (2016) 0.75
Genomic Editing of Non-Coding RNA Genes with CRISPR/Cas9 Ushers in a Potential Novel Approach to Study and Treat Schizophrenia. Front Mol Neurosci (2017) 0.75
To cleave or not to cleave: therapeutic gene editing with and without programmable nucleases. Nat Rev Drug Discov (2017) 0.75
Modulation of human allogeneic and syngeneic pluripotent stem cells and immunological implications for transplantation. Transplant Rev (Orlando) (2016) 0.75
Gene Editing of Human Hematopoietic Stem and Progenitor Cells: Promise and Potential Hurdles. Hum Gene Ther (2016) 0.75
Keeping homologous recombination in check. Cell Res (2016) 0.75
A simple and efficient method to visualize and quantify the efficiency of chromosomal mutations from genome editing. Sci Rep (2016) 0.75
Genome editing: progress and challenges for medical applications. Genome Med (2016) 0.75
Merging Two Strategies for Mixed-Sequence Recognition of Double-Stranded DNA: Pseudocomplementary Invader Probes. J Org Chem (2016) 0.75
Biopharmaceuticals from microorganisms: from production to purification. Braz J Microbiol (2016) 0.75
Emerging cellular and gene therapies for congenital anemias. Am J Med Genet C Semin Med Genet (2016) 0.75
Gene therapy: Gene-editing therapy for neurological disease. Nat Rev Neurol (2016) 0.75
Nrl knockdown by AAV-delivered CRISPR/Cas9 prevents retinal degeneration in mice. Nat Commun (2017) 0.75
Current Biosafety Considerations in Stem Cell Therapy. Cell J (2016) 0.75
Spatial organization of heterologous metabolic system in vivo based on TALE. Sci Rep (2016) 0.75
Molecular Pathways: Breaking the Epithelial Cancer Barrier for Chimeric Antigen Receptor and T-cell Receptor Gene Therapy. Clin Cancer Res (2016) 0.75
ACF7 regulates inflammatory colitis and intestinal wound response by orchestrating tight junction dynamics. Nat Commun (2017) 0.75
Gene suppression approaches to neurodegeneration. Alzheimers Res Ther (2017) 0.75
Ex vivo Generation of Genetically Modified Macrophages from Human Induced Pluripotent Stem Cells. Transfus Med Hemother (2017) 0.75
A lentivirus-free inducible CRISPR-Cas9 system for efficient targeting of human genes. Anal Biochem (2017) 0.75
Genome engineering: a new approach to gene therapy for neuromuscular disorders. Nat Rev Neurol (2017) 0.75
Single-Step qPCR and dPCR Detection of Diverse CRISPR-Cas9 Gene Editing Events in Vivo. G3 (Bethesda) (2017) 0.75
An international effort to cure a global health problem: A report on the 19th Hemoglobin Switching Conference. Exp Hematol (2015) 0.75
Gene Editing and Human Pluripotent Stem Cells: Tools for Advancing Diabetes Disease Modeling and Beta-Cell Development. Curr Diab Rep (2017) 0.75
Therapies targeting DNA and RNA in Huntington's disease. Lancet Neurol (2017) 0.75
The IBD interactome: an integrated view of aetiology, pathogenesis and therapy. Nat Rev Gastroenterol Hepatol (2017) 0.75
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