Unique features of ARCUS nucleases enable high efficiency, targeted gene insertion in vivo

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

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Shoop, W.K., Lape, J., Trum, M. et al. Efficient elimination of MELAS-associated m.3243G mutant mitochondrial DNA by an engineered mitoARCUS nuclease. Nat Metab (2023). https://doi.org/10.1038/s42255-023-00932-6

Preclinical efficacy and safety of ARCUS-POL nucleases for chronic hepatitis B: a potentially curative strategy

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Precise and simultaneous quantification of mitochondrial DNA heteroplasmy and copy number by digital PCR

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Shoop, W. K., Gorsuch, C. L., Bacman, S. R., Moreas, C. T. (2022) Journal of Biological Chemistry, 298(11). https://doi.org/10.1016/j.jbc.2022.102574

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

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Greig, J. A., Breton, C., Ashley, S. N., (2022) Human Gene Therapy. 33(21-22). 1174-1186. http://doi.org/10.1089/hum.2022.061

Targeting Hepatitis B cccDNA with a Sequence-Specific ARCUS Nuclease to Eliminate Hepatitis B Virus In Vivo

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ARCUS gene editing of Apolipoprotein C3 results in substantial reduction in serum triglycerides in vivo

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Shoop W., et al. European Society of Gene & Cell Therapy (ESGCT) 2022 Annual Congress.

ARCUS Gene Editing to Eliminate MELAS-Associated m.3243A>G Mutant Mitochondrial DNA

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Shoop W., et al. American Society of Gene and Cell Therapy (ASGCT) 2022 Annual Meeting.

Targeting the Hepatitis B cccDNA with a Sequence-specific ARCUS Nuclease to Eliminate Hepatitis B Virus

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Targeting the Hepatitis B cccDNA with a Sequence-Specific ARCUS Nuclease to Eliminate Hepatitis B Virus In Vivo

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Gorsuch C. L., Nemec, P., Yu, M., (2022) Molecular Therapy. 30(9), 2909-2022. https://doi.org/10.1016/j.ymthe.2022.05.013

Mitochondrial Targeted Meganuclease as a Platform to Eliminate Mutant mtDNA In Vivo

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Zekonyte, U. Bacman, S. R., Smith, J. (2021) Nature Communications. 12, 3210. https://doi.org/10.1038/s41467-021-23561-7

Translation of an AAV-Delivered Gene Editing Approach for Transthyretin Amyloidosis in Animal Models

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Long-Term Stable Reduction of Low-Density Lipoprotein in Nonhuman Primates Following In Vivo Genome Editing of PCSK9

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Wang, L., Breton, C., Warzecha, C. C., (2021) Molecular Therapy. 6, 2019-2029. https://doi.org/10.1016/j.ymthe.2021.02.020

Meganuclease Targeting of PCSK9 in Macaque Liver Leads to Stable Reduction in Serum Cholesterol

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Wang, L., Smith, J., Breton, C. (2018) Nature Biotechnology. 36, 717–725. https://doi.org/10.1038/nbt.4182

Engineering a Self-inactivating Adeno-associated Virus (AAV) Vector for ARCUS Nuclease Delivery

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A Gene Editing Approach to Eliminate Hepatitis B Virus Using ARCUS Meganucleases

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Development of a Clinical-Grade Meganuclease for Allogeneic CAR T Cell Production​

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Allogeneic CAR T cells with Deoxycytidine Kinase Knockdown Demonstrate Resistance to Fludarabine

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Pires, M. et al. Journal for ImmunoTherapy of Cancer. 9,6 (2021). Society for Immunotherapy of Cancer (SITC) 2021 Annual Meeting.

Allogeneic CAR-T PBCAR0191 with Intensified Lymphodepletion is Highly Active in Subjects with Relapsed/Refractory B-Cell Malignancies

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Shah, B. D., Jacobson, C., Solomon, S. R., (2021) Blood. 138(1). American Society of Hematology (ASH) Annual Meeting. https://doi.org/10.1182/blood-2021-150609

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

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Off the Shelf T Cell Therapies for Hematologic Malignancies

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Initial Findings of the Phase 1 Trial of PBCAR0191, a CD19 Targeted Allogeneic CAR T Cell Therapy

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