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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
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
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
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
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 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
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