Optimization of targeted gene insertion using ARCUS nucleases
Shoop W, et al., Precision Genome Engineering 2023 Conference
Specific elimination of m.3243A>G mutant mitochondrial DNA using mitoARCUS
Shoop W., et al., EuroMIT Conference 2023 Conference
Shifting m.3243A>G heteroplasmy with PBGENE-PMM: Gene editing therapy for primary mitochondrial myopathy
Shoop W, et al., Mitochondrial Medicine – Therapeutic Development 2024 Conference
ARCUS-mediated excision of the “hot spot” region of the human dystrophin gene results in functional improvement in a mouse model of Duchenne muscular dystrophy (DMD)
Owens, G., et al. European Society of Gene and Cell Therapy (ESGCT) 2023 Annual Congress.
Unique features of ARCUS nucleases enable high efficiency, targeted gene insertion in vivo
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
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
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
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
AAV-meganuclease-mediated gene targeting achieves efficient and sustained transduction in newborn and infant macaque liver
Wang, L. (2022) International Conference on Ureagenesis Defects and Allied Conditions.
Treating Transthyretin Amyloidosis via Adeno-Associated Virus Vector Delivery of Meganucleases
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
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
Shoop W., et al. European Society of Gene & Cell Therapy (ESGCT) 2022 Annual Congress.
Specific elimination of m.3243A>G mutant mitochondrial DNA using mitoARCUS in cultured cells and a novel xenograft mouse model
Shoop, W., European Society of Gene & Cell Therapy (ESGCT) 2022 Congress.
Targeting the Hepatitis B cccDNA with a Sequence-Specific ARCUS Nuclease to Eliminate Hepatitis B Virus In Vivo
Nemec P., et al. International HBV Meeting 2022.
AAV-Meganuclease-mediated Gene Targeting Achieves Efficient and Sustained Transduction in Newborn and Infant Macaque Liver
Wang L., (2022) American Society of Gene and Cell Therapy (ASGCT) Annual Meeting.
ARCUS Gene Editing to Eliminate MELAS-Associated m.3243A>G Mutant Mitochondrial DNA
Shoop W., et al. American Society of Gene and Cell Therapy (ASGCT) 2022 Annual Meeting.
Optimization of Hydroxyacidoxidase 1 (HAO1) Targeting ARCUS Nucleases for the Treatment of Primary Hyperoxaluria Type 1 (PH1)
Owens G., 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
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
Gorsuch C. L., Nemec, P., Yu, M., (2022) Molecular Therapy. 30(9), 2909-2022. https://doi.org/10.1016/j.ymthe.2022.05.013
Targeting the Hepatitis B cccDNA with a Sequence-Specific ARCUS Nuclease to Eliminate Hepatitis B Virus In Vivo
Gorsuch, C. et al. HEP DART 2021 Annual Conference.
Mitochondrial Targeted Meganuclease as a Platform to Eliminate Mutant mtDNA In Vivo
Zekonyte, U. Bacman, S. R., Smith, J. (2021) Nature Communications. 12, 3210. https://doi.org/10.1038/s41467-021-23561-7
Rho 1-2 Meganuclease, an Allele-Specific Gene Editing Therapy, Rejuvenates Rod Photoreceptor Structure and Function in a Pig Model of adRP
Jalligampala, A. et al. Association for Research in Vision & Ophthalmology (ARVO) 2021 Meeting.
Translation of an AAV-Delivered Gene Editing Approach for Transthyretin Amyloidosis in Animal Models
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
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
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
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
Gorsuch, C. et al. American Society of Gene and Cell Therapy (ASGCT) 2020 Annual Meeting.
Effective Cell Dose and Functional Attributes of Azercabtagene Zapreleucel (Azer-cel; PBCAR0191) Associated with Allogeneic CAR T-Cell Safety and Efficacy in Patients with Relapsed/Refractory B-Cell Lymphoma
Jacobson, C.A., American Society of Hematology (ASH) 2022 Annual Meeting
Effective Cell Dose and Functional Attributes of Azercabtagene Zapreleucel (azer-cel; PBCAR0191) Associate with Allogeneic CAR T-Cell Safety and Efficacy in Patients with Relapsed/Refractory B-Cell Lymphoma
Jacobson, CA. Dana Farber Cancer Institute. Abstract #2005. American Society of Hematology (ASH) 2022 Annual Meeting.
Development of a Clinical-Grade Meganuclease for Allogeneic CAR T Cell Production
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
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
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
Preliminary Safety and Efficacy of PBCAR0191, an Allogeneic ‘Off-the-Shelf’ CD19-Directed CAR-T for Patients with Relapsed/Refractory (R/R) CD19+ B-ALL
Nitin, J. et al. Blood. 138, 650 (2021). American Society of Hematology (ASH) 2021 Annual Meeting.
Preliminary Safety and Efficacy of PBCAR0191, an Allogeneic, Off-the-Shelf CD19-Targeting CAR-T Product, in Relapsed/Refractory (r/r) CD19+ NHL
Shah, B.D. et al. American Society of Clinical Oncology (ASCO) 2021 Annual Meeting.
Integration of a CD19 CAR Into the TCR Alpha Chain Locus Streamlines Production of Allogeneic Gene-Edited CAR T Cells
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
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
Jacobson C.A. et al. American Society of Hematology (ASH) 2019 Annual Meeting.