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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: porter jj. Sci Rep. 2024 Dec 30;14(1):31791. doi: 10.1038/s41598-024-82766-0. Sci Rep. 2024. PMID: 39738338 Free PMC article.
A cystic fibrosis gene editing approach that is on target.
Porter JJ, Lueck JD. Porter JJ, et al. 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.
56 results