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Chromatin-associated lncRNA-splicing factor condensates regulate hypoxia responsive RNA processing of genes pre-positioned near nuclear speckles.
Song YJ, Shinn MK, Bangru S, Wang Y, Sun Q, Hao Q, Chaturvedi P, Freier SM, Perez-Pinera P, Nelson ER, Belmont AS, Guttman M, Prasanth SG, Kalsotra A, Pappu RV, Prasanth KV. Song YJ, et al. Among authors: perez pinera p. bioRxiv [Preprint]. 2024 Oct 31:2024.10.31.621310. doi: 10.1101/2024.10.31.621310. bioRxiv. 2024. PMID: 39554052 Free PMC article. Preprint.
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: perez pinera p. 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: perez pinera p. 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: perez pinera p. 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.
83 results