Lista de obras - Ciaran M Lee - Dominio Público Uruguay

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

artículo científico publicado en 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.

artículo científico publicado en 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

artículo científico publicado en 2019

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

artículo científico publicado en 2017

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

artículo científico publicado en 2014

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.

artículo científico publicado en 2017

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

artículo científico publicado en 2015

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

artículo científico publicado en 2012

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

artículo científico publicado en 2017

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

artículo científico publicado en 2017

Efficient fdCas9 Synthetic Endonuclease with Improved Specificity for Precise Genome Engineering

artículo científico publicado en 2015

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

artículo científico publicado en 2017

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

article

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

artículo científico publicado en 2017

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

artículo científico publicado en 2019

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

artículo científico publicado en 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

artículo científico publicado en 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

artículo científico publicado en 2016

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

artículo científico publicado en 2020

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

artículo científico publicado en 2018

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

artículo científico publicado en 2018

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

artículo científico publicado en 2015

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

artículo científico publicado en 2021

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

artículo científico publicado en 2016

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

artículo científico publicado en 2021

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

artículo científico publicado en 2019

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

artículo científico publicado en 2016

Lista de obras de Ciaran M Lee

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

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

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

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

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

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

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

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

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

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

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

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

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

Efficient fdCas9 Synthetic Endonuclease with Improved Specificity for Precise Genome Engineering

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

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

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

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

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

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

In Vivo Ryr2 Editing Corrects Catecholaminergic Polymorphic Ventricular Tachycardia

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

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

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

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

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

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

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

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

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

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

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

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