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Mitochondrial targeted meganuclease as a platform to eliminate mutant mtDNA in vivo

Nature communications, 2021-05, Vol.12 (1), p.3210-3210, Article 3210

2021

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Meganuclease targeting of PCSK9 in macaque liver leads to stable reduction in serum cholesterol

Nature biotechnology, 2018-09, Vol.36 (8), p.717-725

2018

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Male‐sterile maize plants produced by targeted mutagenesis of the cytochrome P450‐like gene (MS26) using a re‐designed I–CreI homing endonuclease

The Plant journal : for cell and molecular biology, 2013-12, Vol.76 (5), p.888-899

2013

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Targeted molecular trait stacking in cotton through targeted double‐strand break induction

Plant biotechnology journal, 2013-10, Vol.11 (8), p.933-941

2013

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Cytoplasmic male sterility and abortive seed traits generated through mitochondrial genome editing coupled with allotopic expression of atp1 in tobacco

Frontiers in plant science, 2023-09, Vol.14, p.1253640-1253640

2023

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Probing the DNA-Binding Affinity and Specificity of Designed Zinc Finger Proteins

Biophysical journal, 2010-03, Vol.98 (5), p.852-860

2010

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Targeted DNA excision in Arabidopsis by a re-engineered homing endonuclease

BMC biotechnology, 2012-11, Vol.12 (1), p.86-86, Article 86

2012

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The Design of Functional DNA-Binding Proteins Based on Zinc Finger Domains

Chemical reviews, 2004-02, Vol.104 (2), p.789-800

2004

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Reduction in DNA-binding affinity of Cys2His2 zinc finger proteins by linker phosphorylation

Proceedings of the National Academy of Sciences - PNAS, 2004-05, Vol.101 (20), p.7589-7593

2004

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Long-term stable reduction of low-density lipoprotein in nonhuman primates following in vivo genome editing of PCSK9

Molecular therapy, 2021-06, Vol.29 (6), p.2019-2029

2021

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Integration of a CD19 CAR into the TCR Alpha Chain Locus Streamlines Production of Allogeneic Gene-Edited CAR T Cells

Molecular therapy, 2017-04, Vol.25 (4), p.949-961

2017

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Generation of Rag1‐knockout immunodeficient rats and mice using engineered meganucleases

The FASEB journal, 2013-02, Vol.27 (2), p.703-711

2013

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Targeting the hepatitis B cccDNA with a sequence-specific ARCUS nuclease to eliminate hepatitis B virus in vivo

Molecular therapy, 2022-09, Vol.30 (9), p.2909-2922

2022

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Heritable targeted mutagenesis in maize using a designed endonuclease

The Plant journal : for cell and molecular biology, 2010-01, Vol.61 (1), p.176-187

2010

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Treating Transthyretin Amyloidosis via Adeno-Associated Virus Vector Delivery of Meganucleases

Human gene therapy, 2022-11, Vol.33 (21-22), p.1174

2022

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Efficient elimination of MELAS-associated m.3243G mutant mitochondrial DNA by an engineered mitoARCUS nuclease

Nature metabolism, 2023-12, Vol.5 (12), p.2169-2183

2023

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Transient Expression of Virally Delivered Meganuclease In Planta Generates Inherited Genomic Deletions

Molecular plant, 2015-08, Vol.8 (8), p.1292-1294

2015

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579. Meganucleases as an Efficient Tool for Genome Editing

Molecular therapy, 2016-05, Vol.24, p.S230-S230

2016

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138. Potential Therapeutic Treatment of Friedrich's Ataxia Using Highly Specific Engineered Meganucleases

Molecular therapy, 2016-05, Vol.24, p.S56-S56

2016

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T cell receptor knockout efficiency utilizing an engineered meganuclease is influenced by stimulation conditions

The Journal of immunology (1950), 2016-05, Vol.196 (1_Supplement), p.138-138.2

2016

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394. Generation of a Novel Allogeneic CAR T Cell Platform Utilizing an Engineered Meganuclease and AAV Donor Template to Achieve Efficient Disruption of T Cell Receptor Expression and Simultaneous Homology-Directed Insertion of a CD19 CAR

Molecular therapy, 2016-05, Vol.24, p.S156-S156

2016

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200. Generation of CAR-T Cells Lacking T Cell Receptor and Human Leukocyte Antigen Using Engineered Meganucleases

Molecular therapy, 2016-05, Vol.24, p.S78-S78

2016

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Male‐sterile maize plants produced by targeted mutagenesis of the cytochrome P 450‐like gene ( MS 26 ) using a re‐designed I– C re I homing endonuclease

The Plant journal : for cell and molecular biology, 2013-12, Vol.76 (5), p.888-899

2013

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Mitochondrial targeted meganuclease as a platform to eliminate mutant mtDNA in vivo

Meganuclease targeting of PCSK9 in macaque liver leads to stable reduction in serum cholesterol

Male‐sterile maize plants produced by targeted mutagenesis of the cytochrome P450‐like gene (MS26) using a re‐designed I–CreI homing endonuclease

Targeted molecular trait stacking in cotton through targeted double‐strand break induction

Cytoplasmic male sterility and abortive seed traits generated through mitochondrial genome editing coupled with allotopic expression of atp1 in tobacco

Probing the DNA-Binding Affinity and Specificity of Designed Zinc Finger Proteins

Targeted DNA excision in Arabidopsis by a re-engineered homing endonuclease

The Design of Functional DNA-Binding Proteins Based on Zinc Finger Domains

Reduction in DNA-binding affinity of Cys2His2 zinc finger proteins by linker phosphorylation

Long-term stable reduction of low-density lipoprotein in nonhuman primates following in vivo genome editing of PCSK9

Integration of a CD19 CAR into the TCR Alpha Chain Locus Streamlines Production of Allogeneic Gene-Edited CAR T Cells

OS-034 - A gene editing approach for chronic hepatitis B: elimination of hepatitis B virus in vivo by targeting cccDNA and integrated viral genomes with a sequence-specific ARCUS nuclease

A gene editing approach for chronic hepatitis B: elimination of hepatitis B virus in vivo by targeting cccDNA and integrated viral genomes with a sequence-specific ARCUS nuclease

Generation of Rag1‐knockout immunodeficient rats and mice using engineered meganucleases

Targeting the hepatitis B cccDNA with a sequence-specific ARCUS nuclease to eliminate hepatitis B virus in vivo

Heritable targeted mutagenesis in maize using a designed endonuclease

Treating Transthyretin Amyloidosis via Adeno-Associated Virus Vector Delivery of Meganucleases

Efficient elimination of MELAS-associated m.3243G mutant mitochondrial DNA by an engineered mitoARCUS nuclease

Transient Expression of Virally Delivered Meganuclease In Planta Generates Inherited Genomic Deletions

579. Meganucleases as an Efficient Tool for Genome Editing

138. Potential Therapeutic Treatment of Friedrich's Ataxia Using Highly Specific Engineered Meganucleases

T cell receptor knockout efficiency utilizing an engineered meganuclease is influenced by stimulation conditions

394. Generation of a Novel Allogeneic CAR T Cell Platform Utilizing an Engineered Meganuclease and AAV Donor Template to Achieve Efficient Disruption of T Cell Receptor Expression and Simultaneous Homology-Directed Insertion of a CD19 CAR

200. Generation of CAR-T Cells Lacking T Cell Receptor and Human Leukocyte Antigen Using Engineered Meganucleases

Male‐sterile maize plants produced by targeted mutagenesis of the cytochrome P 450‐like gene ( MS 26 ) using a re‐designed I– C re I homing endonuclease

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