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Single intravitreal administration of a tetravalent siRNA exhibits robust and efficient gene silencing in rodent and swine photoreceptors.
Cheng SY, Caiazzi J, Biscans A, Alterman JF, Echeverria D, McHugh N, Hassler M, Jolly S, Giguere D, Cipi J, Khvorova A, Punzo C. Cheng SY, et al. bioRxiv [Preprint]. 2023 Sep 22:2023.09.20.558641. doi: 10.1101/2023.09.20.558641. bioRxiv. 2023. Update in: Mol Ther Nucleic Acids. 2023 Dec 05;35(1):102088. doi: 10.1016/j.omtn.2023.102088 PMID: 37790464 Free PMC article. Updated. Preprint.
Reduction of retinal ganglion cell death in mouse models of familial dysautonomia using AAV-mediated gene therapy and splicing modulators.
Schultz A, Cheng SY, Kirchner E, Costello S, Miettinen H, Chaverra M, King C, George L, Zhao X, Narasimhan J, Weetall M, Slaugenhaupt S, Morini E, Punzo C, Lefcort F. Schultz A, et al. Among authors: cheng sy. bioRxiv [Preprint]. 2023 May 24:2023.05.22.541535. doi: 10.1101/2023.05.22.541535. bioRxiv. 2023. Update in: Sci Rep. 2023 Oct 30;13(1):18600. doi: 10.1038/s41598-023-45376-w PMID: 37293016 Free PMC article. Updated. Preprint.
1,065 results