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List of works by Ciaran M Lee

A Burden of Rare Variants Associated with Extremes of Gene Expression in Human Peripheral Blood.

scientific article published on February 2016

A Self-Deleting AAV-CRISPR System for Genome Editing

A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells

scientific article published on 06 August 2018

Analysis of gene repair tracts from Cas9/gRNA double-stranded breaks in the human CFTR gene.

scientific article published on 25 August 2016

Author Correction: Gene correction for SCID-X1 in long-term hematopoietic stem cells

article

Author Correction: Gene correction for SCID-X1 in long-term hematopoietic stem cells

scientific article published on 04 December 2019

CD7-edited T cells expressing a CD7-specific CAR for the therapy of T-cell malignancies.

scientific article

COSMID: A Web-based Tool for Identifying and Validating CRISPR/Cas Off-target Sites

scientific article

CRISPR-based gene editing enables FOXP3 gene repair in IPEX patient cells

scientific article published on 06 May 2020

CRISPR/Cas9-Based Genome Editing for Disease Modeling and Therapy: Challenges and Opportunities for Nonviral Delivery.

scientific article

Collagen-rich airway smooth muscle cells are a metastatic niche for tumor colonization in the lung

scientific article published on 13 May 2019

Controlled delivery of β-globin-targeting TALENs and CRISPR/Cas9 into mammalian cells for genome editing using microinjection

scientific article

Correction of the ΔF508 Mutation in the Cystic Fibrosis Transmembrane Conductance Regulator Gene by Zinc-Finger Nuclease Homology-Directed Repair

scientific article published on June 2012

Design and Validation of CRISPR/Cas9 Systems for Targeted Gene Modification in Induced Pluripotent Stem Cells

scientific article published in January 2017

Efficient CRISPR/Cas9-Mediated Genome Editing Using a Chimeric Single-Guide RNA Molecule.

scientific article published on 24 August 2017

Efficient fdCas9 Synthetic Endonuclease with Improved Specificity for Precise Genome Engineering

scientific article published on 30 July 2015

Examination of CRISPR/Cas9 design tools and the effect of target site accessibility on Cas9 activity

scientific article

Gene correction for SCID-X1 in long-term hematopoietic stem cells

article

Genome editing for inborn errors of metabolism: advancing towards the clinic.

scientific article

High-Efficiency, Selection-free Gene Repair in Airway Stem Cells from Cystic Fibrosis Patients Rescues CFTR Function in Differentiated Epithelia

scientific article published on 12 December 2019

Highly efficient editing of the β-globin gene in patient-derived hematopoietic stem and progenitor cells to treat sickle cell disease

scientific article published on 01 September 2019

Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type I

scientific article published on 06 September 2019

In Vivo Ryr2 Editing Corrects Catecholaminergic Polymorphic Ventricular Tachycardia

scientific article published on 01 September 2018

Metabolic engineering generates a transgene-free safety switch for cell therapy

scientific article published on 13 July 2020

Nuclease Target Site Selection for Maximizing On-target Activity and Minimizing Off-target Effects in Genome Editing

scientific article

Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements

scientific article published on 17 October 2018

Site-Specific Post-translational Surface Modification of Adeno-Associated Virus Vectors Using Leucine Zippers

scientific article published on 18 February 2020

Somatic Editing of Ldlr With Adeno-Associated Viral-CRISPR Is an Efficient Tool for Atherosclerosis Research

scientific article published on 01 September 2018

Spatial control of in vivo CRISPR-Cas9 genome editing via nanomagnets

scientific article published on 12 November 2018

Streptococcus thermophilus CRISPR-Cas9 Systems Enable Specific Editing of the Human Genome

scientific article published on 14 December 2015

TNF-α synergises with IFN-γ to induce caspase-8-JAK1/2-STAT1-dependent death of intestinal epithelial cells

scientific article published on 23 September 2021

The Neisseria meningitidis CRISPR-Cas9 System Enables Specific Genome Editing in Mammalian Cells

scientific article published on 19 January 2016

The TRACE-Seq method tracks recombination alleles and identifies clonal reconstitution dynamics of gene targeted human hematopoietic stem cells

scientific article published on 20 January 2021

Therapeutically relevant engraftment of a CRISPR-Cas9-edited HSC-enriched population with HbF reactivation in nonhuman primates

scientific article published on 01 July 2019

Treating hemoglobinopathies using gene-correction approaches: promises and challenges.

scientific article published on 17 June 2016

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