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10.1038/s41467-025-67112-w http://scihub22266oqcxt.onion/10.1038/s41467-025-67112-w 41353207!?!41353207       Nat+Commun 2025 ; ? (?): ? Nephropedia Template TP {{tp|p=41353207|t=2025. CRISPR-free RNA base editing mediated PTC-readthrough restores hearing in mice with Otof nonsense mutation |pdf=|usr=}}{{41353207}} gab.com Text CRISPR-free RNA base editing mediated PTC-readthrough restores hearing in mice with Otof nonsense mutation JO: Nat Commun http://www.kidney.de/pget.php?&tw=1&pmid=41353207 #FOAvip The gene therapy achieved by AAV-mediated otoferlin-overexpression is an effective therapeutic strategy for congenital deafness. However, achieving its physiological and endogenous patterns of expression remains challenging. Here, we generate the homologous mutation Otof c.1315 C > T (p.R439*), equivalent to OTOF c.1273 C > T (p.R425*) found in humans with profound deafness, to create a nonsense mutation-induced deaf mouse model. We then deliver the 'RESTART v3' system, which is a CRISPR-free RNA base editor for nonsense mutation suppression, into the cochlea of the mice. We achieve physiological otoferlin expression, and the edited premature termination codon is reverse-mutated to the original amino acid. We observe significant hearing restoration and enhancement of the behavioral auditory startle reflex. Thus, our study presents a successful RNA editing strategy to significantly restore hereditary deafness in mice carrying the specific Otof nonsense mutation, which holds great promise for future clinical translation. Twit Text FOAVip CRISPR-free RNA base editing mediated PTC-readthrough restores hearing in mice with Otof nonsense mutation ????Nat Commun http://www.kidney.de/pget.php?&tw=1&pmid=41353207 #FOAvip Twit Text # Free Paper on # # # # # LINK http://www.kidney.de/pget.php?&tw=1&pmid=41353207 #FOAvip English Wikipedia <ref>{{Cite pmid|41353207}}</ref> CRISPR-free RNA base editing mediated PTC-readthrough restores hearing in mice with Otof nonsense mutation #MMPMID41353207 Sun H; Teng Q; Liu W; Guo R; Li M; Xiong W; Huang Q; Yu Q; Luo N; Li Y; Song J; Gong S; Shi X; Yi C; Liu K Nat Commun 2025[Dec]; ? (?): ? PMID41353207show ga The gene therapy achieved by AAV-mediated otoferlin-overexpression is an effective therapeutic strategy for congenital deafness. However, achieving its physiological and endogenous patterns of expression remains challenging. Here, we generate the homologous mutation Otof c.1315 C > T (p.R439*), equivalent to OTOF c.1273 C > T (p.R425*) found in humans with profound deafness, to create a nonsense mutation-induced deaf mouse model. We then deliver the 'RESTART v3' system, which is a CRISPR-free RNA base editor for nonsense mutation suppression, into the cochlea of the mice. We achieve physiological otoferlin expression, and the edited premature termination codon is reverse-mutated to the original amino acid. We observe significant hearing restoration and enhancement of the behavioral auditory startle reflex. Thus, our study presents a successful RNA editing strategy to significantly restore hereditary deafness in mice carrying the specific Otof nonsense mutation, which holds great promise for future clinical translation. ?CRISPR-free RNA base editing mediated PTC-readthrough restores hearing(epmc).pdf DeepDyve Pubget Overpricing