Optimization of targeted gene insertion using ARCUS nucleases

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Shoop W, et al., Precision Genome Engineering 2023 Conference

Shifting m.3243A>G heteroplasmy with PBGENE-PMM: Gene editing therapy for primary mitochondrial myopathy

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Shoop W, et al., Mitochondrial Medicine – Therapeutic Development 2024 Conference

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

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Gorsuch, C., et al. European Society of Gene and Cell Therapy (ESGCT) 2023 Annual Congress.

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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Harrison E., et al. American Association for the Study of Liver Diseases (AASLD) The Liver Meeting 2023.

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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Gorsuch, C. L., (2022). European Society of Gene & Cell Therapy (ESGCT) Annual Congress.

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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Gorsuch C. L., (2022) American Society of Gene and Cell Therapy (ASGCT) Annual Meeting.

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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Greig, J.A. et al. American Society of Gene and Cell Therapy (ASGCT) 2021 Annual Meeting.

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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Li, H. et al. American Society of Gene and Cell Therapy (ASGCT) 2020 Annual Meeting.

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

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Gorsuch, C. et al. American Society of Gene and Cell Therapy (ASGCT) 2020 Annual Meeting.

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

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Lape, J. et al. American Society of Gene and Cell Therapy (ASGCT) 2018 Annual Meeting.

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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MacLeod D.T., Antony J., Martin A.J., (2017) Molecular Therapy. 25(4):949-961. https://doi.org/10.1016/j.ymthe.2017.02.005

Off the Shelf T Cell Therapies for Hematologic Malignancies

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McCreedy, B. J., Senyukov, V. V., Nguyen, K. (2018) Best Practice & Research Clinical Haematology. 31(2) 166-175. https://doi.org/10.1016/j.beha.2018.03.001

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

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Jacobson C.A. et al. American Society of Hematology (ASH) 2019 Annual Meeting.