Engineered amphiphilic peptides enable delivery of proteins and CRISPR-associated nucleases to airway epithelia
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journal
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October 2019 |
Meganuclease targeting of PCSK9 in macaque liver leads to stable reduction in serum cholesterol
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journal
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July 2018 |
In vivo gene editing in dystrophic mouse muscle and muscle stem cells
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journal
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December 2015 |
CRISPR-CasΦ from huge phages is a hypercompact genome editor
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journal
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July 2020 |
CRISPR-Edited Stem Cells in a Patient with HIV and Acute Lymphocytic Leukemia
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journal
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September 2019 |
Imaging CAR T cell therapy with PSMA-targeted positron emission tomography
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journal
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July 2019 |
Genome editing with CRISPR–Cas nucleases, base editors, transposases and prime editors
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journal
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June 2020 |
In vivo genome editing restores haemostasis in a mouse model of haemophilia
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journal
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June 2011 |
The delivery challenge: fulfilling the promise of therapeutic genome editing
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journal
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June 2020 |
The chemical evolution of oligonucleotide therapies of clinical utility
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journal
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February 2017 |
Peptide Nucleic Acids and Gene Editing: Perspectives on Structure and Repair
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journal
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February 2020 |
Improving CRISPR Genome Editing by Engineering Guide RNAs
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journal
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August 2019 |
Functional annotation of native enhancers with a Cas9–histone demethylase fusion
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journal
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March 2015 |
Cytosine and adenine base editing of the brain, liver, retina, heart and skeletal muscle of mice via adeno-associated viruses
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journal
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January 2020 |
Radiolabeling Rhesus Monkey CD34 + Hematopoietic and Mesenchymal Stem Cells with 64 Cu-Pyruvaldehyde-Bis(N4-Methylthiosemicarbazone) for MicroPET Imaging
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journal
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January 2008 |
Correction of the Marfan Syndrome Pathogenic FBN1 Mutation by Base Editing in Human Cells and Heterozygous Embryos
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journal
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November 2018 |
Nanoparticles that deliver triplex-forming peptide nucleic acid molecules correct F508del CFTR in airway epithelium
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journal
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April 2015 |
Efficient genome editing in the mouse brain by local delivery of engineered Cas9 ribonucleoprotein complexes
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journal
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February 2017 |
Expression of a site-specific endonuclease stimulates homologous recombination in mammalian cells.
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journal
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June 1994 |
Longitudinal Visualization of Viable Cancer Cell Intratumoral Distribution in Mouse Models Using Oatp1a1-Enhanced Magnetic Resonance Imaging
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journal
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January 2019 |
Gene Therapy
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journal
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August 2019 |
The 4D nucleome project
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journal
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September 2017 |
Nanotechnology for delivery of peptide nucleic acids (PNAs)
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journal
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October 2016 |
Cardiac Chemical Exchange Saturation Transfer MR Imaging Tracking of Cell Survival or Rejection in Mouse Models of Cell Therapy
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journal
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January 2017 |
Advances in genome editing through control of DNA repair pathways
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journal
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December 2019 |
Optimized base editors enable efficient editing in cells, organoids and mice
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journal
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October 2018 |
Structure and Engineering of Francisella novicida Cas9
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journal
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February 2016 |
Prevalence of Pre-existing Antibodies to CRISPR-Associated Nuclease Cas9 in the USA Population
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journal
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September 2018 |
Introducing a Spectrum of Long-Range Genomic Deletions in Human Embryonic Stem Cells Using Type I CRISPR-Cas
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journal
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June 2019 |
A dual-deaminase CRISPR base editor enables concurrent adenine and cytosine editing
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journal
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June 2020 |
A TALE nuclease architecture for efficient genome editing
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journal
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December 2010 |
RNA-programmed genome editing in human cells
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journal
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January 2013 |
Nucleotide-resolution DNA double-strand break mapping by next-generation sequencing
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journal
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March 2013 |
Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy
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journal
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August 2018 |
High prevalence of Streptococcus pyogenes Cas9-reactive T cells within the adult human population
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journal
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October 2018 |
Minimal PAM specificity of a highly similar SpCas9 ortholog
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journal
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October 2018 |
Construction and testing of engineered zinc-finger proteins for sequence-specific modification of mtDNA
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journal
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February 2010 |
The Economics of Reproducibility in Preclinical Research
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journal
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June 2015 |
Mapping the genomic landscape of CRISPR–Cas9 cleavage
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journal
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May 2017 |
Identification of preexisting adaptive immunity to Cas9 proteins in humans
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journal
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January 2019 |
Improved reproducibility by assuring confidence in measurements in biomedical research
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journal
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August 2014 |
Structure-guided chemical modification of guide RNA enables potent non-viral in vivo genome editing
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journal
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November 2017 |
Gene Editing of CCR5 in Autologous CD4 T Cells of Persons Infected with HIV
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journal
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March 2014 |
Base editors for simultaneous introduction of C-to-T and A-to-G mutations
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journal
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June 2020 |
In vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle delivery
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journal
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October 2016 |
CIRCLE-seq: a highly sensitive in vitro screen for genome-wide CRISPR–Cas9 nuclease off-targets
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journal
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May 2017 |
The human body at cellular resolution: the NIH Human Biomolecular Atlas Program
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journal
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January 2019 |
Genome editing to 're-write' wrongs
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journal
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June 2018 |
MitoTALEN reduces mutant mtDNA load and restores tRNAAla levels in a mouse model of heteroplasmic mtDNA mutation
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journal
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September 2018 |
Glycosylase base editors enable C-to-A and C-to-G base changes
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journal
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July 2020 |
Systemic and Persistent Muscle Gene Expression in Rhesus Monkeys with a Liver De-Targeted Adeno-Associated Virus Vector
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journal
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May 2017 |
A bacterial cytidine deaminase toxin enables CRISPR-free mitochondrial base editing
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journal
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July 2020 |
RNA-Guided Human Genome Engineering via Cas9
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journal
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January 2013 |
In vivo base editing restores sensory transduction and transiently improves auditory function in a mouse model of recessive deafness
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journal
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June 2020 |
Efficient genome editing in zebrafish using a CRISPR-Cas system
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journal
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January 2013 |
Superparamagnetic iron oxides as MPI tracers: A primer and review of early applications
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journal
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January 2019 |
Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease
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journal
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January 2013 |
A multifunctional AAV–CRISPR–Cas9 and its host response
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journal
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September 2016 |
Transposon-encoded CRISPR–Cas systems direct RNA-guided DNA integration
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journal
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June 2019 |
RNA-guided editing of bacterial genomes using CRISPR-Cas systems
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journal
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January 2013 |
CasX enzymes comprise a distinct family of RNA-guided genome editors
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journal
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February 2019 |
Delivery technologies for genome editing
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journal
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March 2017 |
Multifunctional CRISPR-Cas9 with engineered immunosilenced human T cell epitopes
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journal
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April 2019 |
New CRISPR–Cas systems from uncultivated microbes
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journal
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December 2016 |
How measurement science can improve confidence in research results
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journal
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April 2018 |
Fetal Gene Transfer Using Lentiviral Vectors: In Vivo Detection of Gene Expression by microPET and Optical Imaging in Fetal and Infant Monkeys
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journal
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December 2006 |
Single-cell bioluminescence imaging of deep tissue in freely moving animals
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journal
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February 2018 |
CRISPR C-to-G base editors for inducing targeted DNA transversions in human cells
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journal
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July 2020 |
A new five-year plan for the U.S. Human Genome Project
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journal
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October 1993 |
Search-and-replace genome editing without double-strand breaks or donor DNA
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journal
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October 2019 |
Therapeutic base editing of human hematopoietic stem cells
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journal
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March 2020 |
Beyond editing: repurposing CRISPR–Cas9 for precision genome regulation and interrogation
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journal
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December 2015 |
Adenine base editing in mouse embryos and an adult mouse model of Duchenne muscular dystrophy
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journal
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April 2018 |
Mitochondrial Genome Engineering: The Revolution May Not Be CRISPR-Ized
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journal
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February 2018 |
MitoTALEN: A General Approach to Reduce Mutant mtDNA Loads and Restore Oxidative Phosphorylation Function in Mitochondrial Diseases
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journal
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October 2015 |
Adenine base editing in an adult mouse model of tyrosinaemia
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journal
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February 2019 |
Lentiviral vectors, two decades later
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journal
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September 2016 |
Immune-orthogonal orthologues of AAV capsids and of Cas9 circumvent the immune response to the administration of gene therapy
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journal
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July 2019 |
A thermostable Cas9 with increased lifetime in human plasma
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journal
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November 2017 |
Radiolabeling and In Vivo Imaging of Transplanted Renal Lineages Differentiated from Human Embryonic Stem Cells in Fetal Rhesus Monkeys
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journal
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April 2011 |
An unbiased genome-wide analysis of zinc-finger nuclease specificity
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journal
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August 2011 |
Long-Term Luciferase Expression Monitored by Bioluminescence Imaging After Adeno-Associated Virus-Mediated Fetal Gene Delivery in Rhesus Monkeys ( Macaca mulatta )
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journal
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February 2010 |
A New Class of Medicines through DNA Editing
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journal
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March 2019 |
Functionally diverse type V CRISPR-Cas systems
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journal
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December 2018 |
Versatile and robust genome editing with Streptococcus thermophilus CRISPR1-Cas9
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journal
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January 2020 |
Evolutionary classification of CRISPR–Cas systems: a burst of class 2 and derived variants
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journal
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December 2019 |
A Compact, High-Accuracy Cas9 with a Dinucleotide PAM for In Vivo Genome Editing
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journal
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February 2019 |
The NextGen Genetic Association Studies Consortium: A Foray into In Vitro Population Genetics
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journal
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April 2017 |
Molecular remission of infant B-ALL after infusion of universal TALEN gene-edited CAR T cells
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journal
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January 2017 |
Genome editing in mitochondria corrects a pathogenic mtDNA mutation in vivo
|
journal
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September 2018 |
Efficient genome engineering in human pluripotent stem cells using Cas9 from Neisseria meningitidis
|
journal
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August 2013 |
In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy
|
journal
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December 2015 |
RNA-guided DNA insertion with CRISPR-associated transposases
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journal
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June 2019 |
CHANGE-seq reveals genetic and epigenetic effects on CRISPR–Cas9 genome-wide activity
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journal
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June 2020 |
A combinatorial approach to create artificial homing endonucleases cleaving chosen sequences
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journal
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November 2006 |
Radiolabeling Human Peripheral Blood Stem Cells for Positron Emission Tomography (PET) Imaging in Young Rhesus Monkeys
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journal
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October 2013 |
Engineered skeletal muscles for disease modeling and drug discovery
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journal
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November 2019 |
Development and application of human skeletal muscle microphysiological systems
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journal
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January 2018 |
Highly Parallel Profiling of Cas9 Variant Specificity
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journal
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May 2020 |
Synthetic transcription factors for cell fate reprogramming
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journal
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October 2018 |
The promise and challenge of therapeutic genome editing
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journal
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February 2020 |
Allele-selective transcriptional repression of mutant HTT for the treatment of Huntington’s disease
|
journal
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July 2019 |
The International Human Epigenome Consortium: A Blueprint for Scientific Collaboration and Discovery
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journal
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November 2016 |
Orthogonal Cas9 proteins for RNA-guided gene regulation and editing
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journal
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September 2013 |
The next generation of CRISPR–Cas technologies and applications
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journal
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May 2019 |
Adenovirus-Mediated Somatic Genome Editing of Pten by CRISPR/Cas9 in Mouse Liver in Spite of Cas9-Specific Immune Responses
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journal
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July 2015 |
Editing the Epigenome: Reshaping the Genomic Landscape
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journal
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August 2018 |
Non-invasive detection of adeno-associated viral gene transfer using a genetically encoded CEST-MRI reporter gene in the murine heart
|
journal
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March 2018 |
Genome-editing Technologies for Gene and Cell Therapy
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journal
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March 2016 |
Lessons from modENCODE
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journal
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August 2015 |
A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing
|
journal
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February 2018 |
Cpf1 Is a Single RNA-Guided Endonuclease of a Class 2 CRISPR-Cas System
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journal
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October 2015 |
Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy
|
journal
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February 2019 |
A compact Cas9 ortholog from Staphylococcus Auricularis (SauriCas9) expands the DNA targeting scope
|
journal
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March 2020 |
Efficient Generation and Correction of Mutations in Human iPS Cells Utilizing mRNAs of CRISPR Base Editors and Prime Editors
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journal
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May 2020 |
A biodegradable nanocapsule delivers a Cas9 ribonucleoprotein complex for in vivo genome editing
|
journal
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September 2019 |
Cas9-crRNA ribonucleoprotein complex mediates specific DNA cleavage for adaptive immunity in bacteria
|
journal
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September 2012 |
CRISPR-engineered T cells in patients with refractory cancer
|
journal
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February 2020 |
AAV-CRISPR Gene Editing Is Negated by Pre-existing Immunity to Cas9
|
journal
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June 2020 |
Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo
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journal
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February 2016 |
Epigenome editing by a CRISPR-Cas9-based acetyltransferase activates genes from promoters and enhancers
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journal
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April 2015 |
Increasing the efficiency of homology-directed repair for CRISPR-Cas9-induced precise gene editing in mammalian cells
|
journal
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March 2015 |
A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity
|
journal
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June 2012 |
BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis
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journal
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September 2015 |
Somatic gene editing ameliorates skeletal and cardiac muscle failure in pig and human models of Duchenne muscular dystrophy
|
journal
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January 2020 |
Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10
|
journal
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January 2019 |
Evaluating and Enhancing Target Specificity of Gene-Editing Nucleases and Deaminases
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journal
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June 2019 |
Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage
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journal
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April 2016 |
Dual base editor catalyzes both cytosine and adenine base conversions in human cells
|
journal
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June 2020 |
Tracking genome engineering outcome at individual DNA breakpoints
|
journal
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July 2011 |
Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy
|
journal
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November 2017 |
Highly efficient endogenous human gene correction using designed zinc-finger nucleases
|
journal
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April 2005 |
Safety Evaluation of Lipid Nanoparticle–Formulated Modified mRNA in the Sprague-Dawley Rat and Cynomolgus Monkey
|
journal
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November 2017 |
Efficient in vivo gene editing using ribonucleoproteins in skin stem cells of recessive dystrophic epidermolysis bullosa mouse model
|
journal
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January 2017 |
Digenome-seq: genome-wide profiling of CRISPR-Cas9 off-target effects in human cells
|
journal
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February 2015 |
Multiplex Genome Engineering Using CRISPR/Cas Systems
|
journal
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January 2013 |
Defining CRISPR–Cas9 genome-wide nuclease activities with CIRCLE-seq
|
journal
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October 2018 |
Base editing: precision chemistry on the genome and transcriptome of living cells
|
journal
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October 2018 |
In vivo genome editing using Staphylococcus aureus Cas9
|
journal
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April 2015 |
GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases
|
journal
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December 2014 |
CRISPR-Based Therapeutic Genome Editing: Strategies and In Vivo Delivery by AAV Vectors
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journal
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April 2020 |
Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage
|
journal
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October 2017 |
Treatment of a metabolic liver disease by in vivo genome base editing in adult mice
|
journal
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October 2018 |
CRISPR–Cas9-mediated base-editing screening in mice identifies DND1 amino acids that are critical for primordial germ cell development
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journal
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October 2018 |