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A cystic fibrosis gene editing approach that is on target.
Porter JJ, Lueck JD. Porter JJ, et al. Among authors: lueck jd. Mol Ther Nucleic Acids. 2024 May 8;35(2):102197. doi: 10.1016/j.omtn.2024.102197. eCollection 2024 Jun 11. Mol Ther Nucleic Acids. 2024. PMID: 38766524 Free PMC article. No abstract available.
Engineered tRNAs efficiently suppress CDKL5 premature termination codons.
Pezzini S, Mustaccia A, Aboa P, Faustini G, Branchini A, Pinotti M, Frasca A, Porter JJ, Lueck JD, Landsberger N. Pezzini S, et al. Among authors: lueck jd. Sci Rep. 2024 Dec 30;14(1):31791. doi: 10.1038/s41598-024-82766-0. Sci Rep. 2024. PMID: 39738338 Free PMC article.
Gene, RNA, and ASO-based therapeutic approaches in Cystic Fibrosis.
Allaire NE, Griesenbach U, Kerem B, Lueck JD, Stanleigh N, Oren YS. Allaire NE, et al. Among authors: lueck jd. J Cyst Fibros. 2023 Mar;22 Suppl 1(Suppl 1):S39-S44. doi: 10.1016/j.jcf.2022.12.016. Epub 2023 Jan 17. J Cyst Fibros. 2023. PMID: 36658041 Free PMC article.
40 results