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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. 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: shirguppe s. 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. Among authors: shirguppe s. 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.
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: shirguppe s. Cell Discov. 2019 Aug 20;5:41. doi: 10.1038/s41421-019-0109-7. eCollection 2019. Cell Discov. 2019. PMID: 31636954 Free PMC article.