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Page 1
Single-Stranded DNA with Internal Base Modifications Mediates Highly Efficient Gene Insertion in Primary Cells.
Kanke KL, Rayner RE, Abel E, Venugopalan A, Suu M, Stack JT, Nouri R, Guo G, Vetter TA, Cormet-Boyaka E, Hester ME, Vaidyanathan S. Kanke KL, et al. Among authors: vetter ta. bioRxiv [Preprint]. 2024 Feb 1:2024.02.01.578476. doi: 10.1101/2024.02.01.578476. bioRxiv. 2024. Update in: Nucleic Acids Res. 2024 Dec 11;52(22):13561-13576. doi: 10.1093/nar/gkae1069 PMID: 38352420 Free PMC article. Updated. Preprint.
Combination AAV therapy with galectin-1 and SOD1 downregulation demonstrates superior therapeutic effect in a severe ALS mouse model.
Baird MC, Likhite SB, Vetter TA, Caporale JR, Girard HB, Roussel FS, Howard AE, Schwartz MK, Reed AR, Kaleem A, Zhang X, Meyer KC. Baird MC, et al. Among authors: vetter ta. Mol Ther Methods Clin Dev. 2024 Aug 6;32(3):101312. doi: 10.1016/j.omtm.2024.101312. eCollection 2024 Sep 12. Mol Ther Methods Clin Dev. 2024. PMID: 39257530 Free PMC article.
DNA-PKcs Inhibition Improves Sequential Gene Insertion of the Full-Length CFTR cDNA in Airway Stem Cells.
Stack JT, Rayner RE, Nouri R, Suarez CJ, Kim SH, Kanke KL, Vetter TA, Cormet-Boyaka E, Vaidyanathan S. Stack JT, et al. Among authors: vetter ta. bioRxiv [Preprint]. 2024 Aug 12:2024.08.12.607571. doi: 10.1101/2024.08.12.607571. bioRxiv. 2024. Update in: Mol Ther Nucleic Acids. 2024 Sep 16;35(4):102339. doi: 10.1016/j.omtn.2024.102339 PMID: 39185207 Free PMC article. Updated. Preprint.
Evaluation of safety and early efficacy of AAV gene therapy in mouse models of vanishing white matter disease.
Herstine JA, Chang PK, Chornyy S, Stevenson TJ, Sunshine AC, Nokhrina K, Rediger J, Wentz J, Vetter TA, Scholl E, Holaway C, Pyne NK, Bratasz A, Yeoh S, Flanigan KM, Bonkowsky JL, Bradbury AM. Herstine JA, et al. Among authors: vetter ta. Mol Ther. 2024 Jun 5;32(6):1701-1720. doi: 10.1016/j.ymthe.2024.03.034. Epub 2024 Mar 27. Mol Ther. 2024. PMID: 38549375
Prothrombin Knockdown Protects Podocytes and Reduces Proteinuria in Glomerular Disease.
Waller AP, Wolfgang KJ, Pruner I, Stevenson ZS, Abdelghani E, Muralidharan K, Wilkie TK, Blissett AR, Calomeni EP, Vetter TA, Brodsky SV, Smoyer WE, Nieman MT, Kerlin BA. Waller AP, et al. Among authors: vetter ta. bioRxiv [Preprint]. 2024 Mar 2:2023.06.20.544360. doi: 10.1101/2023.06.20.544360. bioRxiv. 2024. PMID: 38464017 Free PMC article. Preprint.
21 results