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Page 1
In vivo CRISPR base editing for treatment of Huntington's disease.
Shirguppe S, Gapinske M, Swami D, Gosstola N, Acharya P, Miskalis A, Joulani D, Szkwarek MG, Bhattacharjee A, Elias G, Stilger M, Winter J, Woods WS, Anand D, Lim CKW, Gaj T, Perez-Pinera P. Shirguppe S, et al. Among authors: gapinske m. bioRxiv [Preprint]. 2024 Jul 6:2024.07.05.602282. doi: 10.1101/2024.07.05.602282. bioRxiv. 2024. PMID: 39005280 Free PMC article. Preprint.
SPLICER: A Highly Efficient Base Editing Toolbox That Enables In Vivo Therapeutic Exon Skipping.
Miskalis A, Shirguppe S, Winter J, Elias G, Swami D, Nambiar A, Stilger M, Woods WS, Gosstola N, Gapinske M, Zeballos A, Moore H, Maslov S, Gaj T, Perez-Pinera P. Miskalis A, et al. Among authors: gapinske m. bioRxiv [Preprint]. 2024 Apr 2:2024.04.01.587650. doi: 10.1101/2024.04.01.587650. bioRxiv. 2024. Update in: Nat Commun. 2024 Nov 28;15(1):10354. doi: 10.1038/s41467-024-54529-y PMID: 38883727 Free PMC article. Updated. Preprint.
Targeting Duchenne muscular dystrophy by skipping DMD exon 45 with base editors.
Gapinske M, Winter J, Swami D, Gapinske L, Woods WS, Shirguppe S, Miskalis A, Busza A, Joulani D, Kao CJ, Kostan K, Bigot A, Bashir R, Perez-Pinera P. Gapinske M, et al. Mol Ther Nucleic Acids. 2023 Jul 27;33:572-586. doi: 10.1016/j.omtn.2023.07.029. eCollection 2023 Sep 12. Mol Ther Nucleic Acids. 2023. PMID: 37637209 Free PMC article.
Treatment of a Mouse Model of ALS by In Vivo Base Editing.
Lim CKW, Gapinske M, Brooks AK, Woods WS, Powell JE, Zeballos C MA, Winter J, Perez-Pinera P, Gaj T. Lim CKW, et al. Among authors: gapinske m. Mol Ther. 2020 Apr 8;28(4):1177-1189. doi: 10.1016/j.ymthe.2020.01.005. Epub 2020 Jan 14. Mol Ther. 2020. PMID: 31991108 Free PMC article.
Targeted exon skipping with AAV-mediated split adenine base editors.
Winter J, Luu A, Gapinske M, Manandhar S, Shirguppe S, Woods WS, Song JS, Perez-Pinera P. Winter J, et al. Among authors: gapinske m. Cell Discov. 2019 Aug 20;5:41. doi: 10.1038/s41421-019-0109-7. eCollection 2019. Cell Discov. 2019. PMID: 31636954 Free PMC article.
Disruption of the β1L Isoform of GABP Reverses Glioblastoma Replicative Immortality in a TERT Promoter Mutation-Dependent Manner.
Mancini A, Xavier-Magalhães A, Woods WS, Nguyen KT, Amen AM, Hayes JL, Fellmann C, Gapinske M, McKinney AM, Hong C, Jones LE, Walsh KM, Bell RJA, Doudna JA, Costa BM, Song JS, Perez-Pinera P, Costello JF. Mancini A, et al. Among authors: gapinske m. Cancer Cell. 2018 Sep 10;34(3):513-528.e8. doi: 10.1016/j.ccell.2018.08.003. Cancer Cell. 2018. PMID: 30205050 Free PMC article.
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