Gene Editing

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  Subject Areas on Research

  • ^* CRISPR-Based Epigenome Editing of Cytokine Receptors for the Promotion of Cell Survival and Tissue Deposition in Inflammatory Environments. 
  • A Decade of CRISPR-Cas Gnome Editing in C. elegans . 
  • AAV CRISPR editing rescues cardiac and muscle function for 18 months in dystrophic mice. 
  • AAV9 Edits Muscle Stem Cells in Normal and Dystrophic Adult Mice. 
  • Academies' action plan for germline editing. 
  • An engineered ScCas9 with broad PAM range and high specificity and activity. 
  • Applications of Gene Editing Technologies to Cellular Therapies. 
  • Breakthrough Moments: Genome Editing and Organoids. 
  • CRISPR genome editing in stem cells turns to gold. 
  • CRISPR-Cas9 epigenome editing enables high-throughput screening for functional regulatory elements in the human genome. 
  • CRISPR-Cas9-Guided Genome Engineering in C. elegans. 
  • CRISPR-Cas9-Guided Genome Engineering in Caenorhabditis elegans. 
  • CRISPR-SID: Identifying EZH2 as a druggable target for desmoid tumors via in vivo dependency mapping. 
  • CRISPR/Cas9 Editing of Murine Induced Pluripotent Stem Cells for Engineering Inflammation-Resistant Tissues. 
  • Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models. 
  • Colonoscopy-based colorectal cancer modeling in mice with CRISPR-Cas9 genome editing and organoid transplantation. 
  • Creation of versatile cloning platforms for transgene expression and dCas9-based epigenome editing. 
  • Cross-species evolution of a highly potent AAV variant for therapeutic gene transfer and genome editing. 
  • Downregulation of SNCA Expression by Targeted Editing of DNA Methylation: A Potential Strategy for Precision Therapy in PD. 
  • Editing the Epigenome: Reshaping the Genomic Landscape. 
  • Editing the Neuronal Genome: a CRISPR View of Chromatin Regulation in Neuronal Development, Function, and Plasticity. 
  • Efficient generation of targeted and controlled mutational events in porcine cells using nuclease-directed homologous recombination. 
  • Emerging approaches and technologies in transplantation: the potential game changers. 
  • Encapsulating Cas9 into extracellular vesicles by protein myristoylation. 
  • Engineering Delivery Vehicles for Genome Editing. 
  • Epigenome editing of the CFTR-locus for treatment of cystic fibrosis. 
  • From CRISPR scissors to virus sensors. 
  • Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy. 
  • Gene Therapy 2017: Progress and Future Directions. 
  • Gene therapy beyond luxturna: a new horizon of the treatment for inherited retinal disease. 
  • Gene therapy for glycogen storage diseases. 
  • Generation and comparison of CRISPR-Cas9 and Cre-mediated genetically engineered mouse models of sarcoma. 
  • Genetic engineering: Chemical control for CRISPR editing. 
  • Genome Engineering for Personalized Arthritis Therapeutics. 
  • Genome Engineering of Stem Cells for Autonomously Regulated, Closed-Loop Delivery of Biologic Drugs. 
  • Genome engineering: a new approach to gene therapy for neuromuscular disorders. 
  • Genome-editing Technologies for Gene and Cell Therapy. 
  • Immunity to Cas9 as an Obstacle to Persistent Genome Editing. 
  • In Situ Gene Therapy via AAV-CRISPR-Cas9-Mediated Targeted Gene Regulation. 
  • In vivo genome editing and organoid transplantation models of colorectal cancer and metastasis. 
  • Increasing the specificity of CRISPR systems with engineered RNA secondary structures. 
  • Induced cardiomyocyte maturation: Cardiac transcription factors are necessary but not sufficient. 
  • Integrating Biomaterials and Genome Editing Approaches to Advance Biomedical Science. 
  • Lentiviral Vector Platform for the Efficient Delivery of Epigenome-editing Tools into Human Induced Pluripotent Stem Cell-derived Disease Models. 
  • Lentiviral Vectors for Delivery of Gene-Editing Systems Based on CRISPR/Cas: Current State and Perspectives. 
  • Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy. 
  • Manifold roles of β-arrestins in GPCR signaling elucidated with siRNA and CRISPR/Cas9. 
  • Mapping of pseudouridine residues on cellular and viral transcripts using a novel antibody-based technique. 
  • Minimal PAM specificity of a highly similar SpCas9 ortholog. 
  • Moving away from one disease at a time: Screening, trial design, and regulatory implications of novel platform technologies. 
  • Myogenic Progenitor Cell Lineage Specification by CRISPR/Cas9-Based Transcriptional Activators. 
  • Optimization of Meniscus Cell Transduction Using Lentivirus and Adeno-Associated Virus for Gene Editing and Tissue Engineering Applications. 
  • Plug-and-Play Protein Modification Using Homology-Independent Universal Genome Engineering. 
  • RGEN Editing of RNA and DNA: The Long and Winding Road from Catalytic RNAs to CRISPR to the Clinic. 
  • Racing for Academic Glory and Patents: Lessons from CRISPR 
  • Rapid Disruption of Genes Specifically in Livers of Mice Using Multiplex CRISPR/Cas9 Editing. 
  • Rapid and reversible epigenome editing by endogenous chromatin regulators. 
  • Reactions to the National Academies/Royal Society Report on Heritable Human Genome Editing . 
  • Reprogramming metabolic pathways in vivo with CRISPR/Cas9 genome editing to treat hereditary tyrosinaemia. 
  • Retinal Ganglion Cells With a Glaucoma OPTN(E50K) Mutation Exhibit Neurodegenerative Phenotypes when Derived from Three-Dimensional Retinal Organoids. 
  • Safe and Effective In Vivo Targeting and Gene Editing in Hematopoietic Stem Cells: Strategies for Accelerating Development. 
  • Screening Regulatory Element Function with CRISPR/Cas9-based Epigenome Editing. 
  • Somatic Liver Knockout (SLiK): A Quick and Efficient Way to Generate Liver-Specific Knockout Mice Using Multiplex CRISPR/Cas9 Gene Editing. 
  • Step-Wise Chondrogenesis of Human Induced Pluripotent Stem Cells and Purification Via a Reporter Allele Generated by CRISPR-Cas9 Genome Editing. 
  • The NIH Somatic Cell Genome Editing program. 
  • The next generation of CRISPR-Cas technologies and applications. 
  • The once and future gene therapy. 
  • These Are the Genes You're Looking For: Finding Host Resistance Genes. 
  • Transgenic mice for in vivo epigenome editing with CRISPR-based systems. 
  • Unwinding the Role of FACT in Cas9-based Genome Editing. 
  • Use of Primary Cultured Hippocampal Neurons to Study the Assembly of Axon Initial Segments. 
  • Viable Mice with Extensive Gene Humanization (25-kbp) Created Using Embryonic Stem Cell/Blastocyst and CRISPR/Zygote Injection Approaches. 
  • Wake-up call from Hong Kong. 
  • Wnt-regulated lncRNA discovery enhanced by in vivo identification and CRISPRi functional validation.