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Spliceosome-Mediated Pre-mRNA trans-Splicing Can Repair CEP290 mRNA.
Dooley SJ, McDougald DS, Fisher KJ, Bennicelli JL, Mitchell LG, Bennett J. Dooley SJ, et al. Mol Ther Nucleic Acids. 2018 Sep 7;12:294-308. doi: 10.1016/j.omtn.2018.05.014. Epub 2018 Jun 27. Mol Ther Nucleic Acids. 2018. PMID: 30195768 Free PMC article.
Amelioration of Neurosensory Structure and Function in Animal and Cellular Models of a Congenital Blindness.
Song JY, Aravand P, Nikonov S, Leo L, Lyubarsky A, Bennicelli JL, Pan J, Wei Z, Shpylchak I, Herrera P, Bennett DJ, Commins N, Maguire AM, Pham J, den Hollander AI, Cremers FPM, Koenekoop RK, Roepman R, Nishina P, Zhou S, Pan W, Ying GS, Aleman TS, de Melo J, McNamara I, Sun J, Mills J, Bennett J. Song JY, et al. Mol Ther. 2018 Jun 6;26(6):1581-1593. doi: 10.1016/j.ymthe.2018.03.015. Epub 2018 Mar 21. Mol Ther. 2018. PMID: 29673930 Free PMC article.
97 results