about
Nuclease Target Site Selection for Maximizing On-target Activity and Minimizing Off-target Effects in Genome EditingCOSMID: A Web-based Tool for Identifying and Validating CRISPR/Cas Off-target SitesEfficient fdCas9 Synthetic Endonuclease with Improved Specificity for Precise Genome EngineeringControlled delivery of β-globin-targeting TALENs and CRISPR/Cas9 into mammalian cells for genome editing using microinjectionGenome editing for inborn errors of metabolism: advancing towards the clinic.Examination of CRISPR/Cas9 design tools and the effect of target site accessibility on Cas9 activity.A Burden of Rare Variants Associated with Extremes of Gene Expression in Human Peripheral Blood.Streptococcus thermophilus CRISPR-Cas9 Systems Enable Specific Editing of the Human GenomeThe Neisseria meningitidis CRISPR-Cas9 System Enables Specific Genome Editing in Mammalian CellsCD7-edited T cells expressing a CD7-specific CAR for the therapy of T-cell malignancies.Treating hemoglobinopathies using gene-correction approaches: promises and challenges.CRISPR/Cas9-Based Genome Editing for Disease Modeling and Therapy: Challenges and Opportunities for Nonviral Delivery.Analysis of gene repair tracts from Cas9/gRNA double-stranded breaks in the human CFTR gene.Efficient CRISPR/Cas9-Mediated Genome Editing Using a Chimeric Single-Guide RNA Molecule.Correction of the ΔF508 Mutation in the Cystic Fibrosis Transmembrane Conductance Regulator Gene by Zinc-Finger Nuclease Homology-Directed Repair.Design and Validation of CRISPR/Cas9 Systems for Targeted Gene Modification in Induced Pluripotent Stem Cells.In Vivo Ryr2 Editing Corrects Catecholaminergic Polymorphic Ventricular TachycardiaA Self-Deleting AAV-CRISPR System for Genome EditingCollagen-rich airway smooth muscle cells are a metastatic niche for tumor colonization in the lungGene correction for SCID-X1 in long-term hematopoietic stem cellsAuthor Correction: Gene correction for SCID-X1 in long-term hematopoietic stem cellsSite-Specific Post-translational Surface Modification of Adeno-Associated Virus Vectors Using Leucine ZippersSomatic Editing of Ldlr With Adeno-Associated Viral-CRISPR Is an Efficient Tool for Atherosclerosis ResearchA high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cellsAuthor Correction: Gene correction for SCID-X1 in long-term hematopoietic stem cellsHigh-Efficiency, Selection-free Gene Repair in Airway Stem Cells from Cystic Fibrosis Patients Rescues CFTR Function in Differentiated EpitheliaTherapeutically relevant engraftment of a CRISPR-Cas9-edited HSC-enriched population with HbF reactivation in nonhuman primatesHighly efficient editing of the β-globin gene in patient-derived hematopoietic stem and progenitor cells to treat sickle cell diseaseSpatial control of in vivo CRISPR-Cas9 genome editing via nanomagnetsOptimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic RearrangementsHuman genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type ICRISPR-based gene editing enables FOXP3 gene repair in IPEX patient cells
P50
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P50
description
hulumtues
@sq
researcher
@en
wetenschapper
@nl
հետազոտող
@hy
name
Ciaran M Lee
@ast
Ciaran M Lee
@en
Ciaran M Lee
@es
Ciaran M Lee
@nl
Ciaran M Lee
@sl
type
label
Ciaran M Lee
@ast
Ciaran M Lee
@en
Ciaran M Lee
@es
Ciaran M Lee
@nl
Ciaran M Lee
@sl
prefLabel
Ciaran M Lee
@ast
Ciaran M Lee
@en
Ciaran M Lee
@es
Ciaran M Lee
@nl
Ciaran M Lee
@sl
P108
P106
P21
P31
P496
0000-0002-0789-9149
P569
2000-01-01T00:00:00Z