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Efficient elimination of MELAS-associated m.3243G mutant mitochondrial DNA by an engineered mitoARCUS nuclease.
Nat Metab. 2023 Dec;5(12):2169-2183. doi: 10.1038/s42255-023-00932-6. Epub 2023 Nov 30.
Nat Metab. 2023.
PMID: 38036771
Free PMC article.
Mitochondrial targeted meganuclease as a platform to eliminate mutant mtDNA in vivo.
Zekonyte U, Bacman SR, Smith J, Shoop W, Pereira CV, Tomberlin G, Stewart J, Jantz D, Moraes CT.
Zekonyte U, et al.
Nat Commun. 2021 May 28;12(1):3210. doi: 10.1038/s41467-021-23561-7.
Nat Commun. 2021.
PMID: 34050192
Free PMC article.
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Targeting the hepatitis B cccDNA with a sequence-specific ARCUS nuclease to eliminate hepatitis B virus in vivo.
Gorsuch CL, Nemec P, Yu M, Xu S, Han D, Smith J, Lape J, van Buuren N, Ramirez R, Muench RC, Holdorf MM, Feierbach B, Falls G, Holt J, Shoop W, Sevigny E, Karriker F, Brown RV, Joshi A, Goodwin T, Tam YK, Lin PJC, Semple SC, Leatherbury N, Delaney Iv WE, Jantz D, Rhoden Smith A.
Gorsuch CL, et al.
Mol Ther. 2022 Sep 7;30(9):2909-2922. doi: 10.1016/j.ymthe.2022.05.013. Epub 2022 May 16.
Mol Ther. 2022.
PMID: 35581938
Free PMC article.
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Precise and simultaneous quantification of mitochondrial DNA heteroplasmy and copy number by digital PCR.
Shoop WK, Gorsuch CL, Bacman SR, Moraes CT.
Shoop WK, et al.
J Biol Chem. 2022 Nov;298(11):102574. doi: 10.1016/j.jbc.2022.102574. Epub 2022 Oct 6.
J Biol Chem. 2022.
PMID: 36209825
Free PMC article.
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Treating Transthyretin Amyloidosis via Adeno-Associated Virus Vector Delivery of Meganucleases.
Greig JA, Breton C, Ashley SN, Martins KM, Gorsuch C, Chorazeczewski JK, Furmanak T, Smith MK, Zhu Y, Bell P, Shoop W, Li H, Smith J, Tomberlin G, Clark P, Mitchell TW, Buza EL, Yan H, Jantz D, Wilson JM.
Greig JA, et al.
Hum Gene Ther. 2022 Nov;33(21-22):1174-1186. doi: 10.1089/hum.2022.061.
Hum Gene Ther. 2022.
PMID: 36375122
Free PMC article.
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