Gene Editing

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.

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.

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.

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.