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Paper

Marasco, Luciano E, Dujardin, Gwendal, Sousa-Luís, Rui, Liu, Ying Hsiu, Stigliano, Jose N, Nomakuchi, Tomoki, Proudfoot, Nick J, Krainer, Adrian R, Kornblihtt, Alberto R (June 2022) Counteracting chromatin effects of a splicing-correcting antisense oligonucleotide improves its therapeutic efficacy in spinal muscular atrophy. Cell, 185 (12). 2057-2070.e15. ISSN 0092-8674

Kim, Young Jin, Nomakuchi, Tomoki, Papaleonidopoulou, Foteini, Yang, Lucia, Zhang, Qian, Krainer, Adrian R (May 2022) Gene-specific nonsense-mediated mRNA decay targeting for cystic fibrosis therapy. Nature Communications, 13 (1). p. 2978. ISSN 2041-1723

Kim, Young Jin, Nomakuchi, Tomoki, Papaleonidopoulou, Foteini, Krainer, Adrian (July 2021) Gene-Specific Nonsense-Mediated mRNA Decay Targeting for Cystic Fibrosis Therapy. BioRxiv. (Unpublished)

Marasco, Luciano, Dujardin, Gwendal, Sousa-Luís, Rui, Liu, Ying Hsiu, Stigliano, José, Nomakuchi, Tomoki, Proudfoot, Nick, Krainer, Adrian, Kornblihtt, Alberto (2021) Towards a combined therapy for spinal muscular atrophy based on opposing effects of an antisense oligonucleotide on chromatin and splicing. bioRxiv. (Submitted)

Sinha, R., Kim, Y. J., Nomakuchi, T., Sahashi, K., Hua, Y., Rigo, F., Bennett, C. F., Krainer, A. R. (June 2018) Antisense oligonucleotides correct the familial dysautonomia splicing defect in IKBKAP transgenic mice. Nucleic Acids Res, 46 (10). pp. 4833-4844. ISSN 0305-1048

Aznarez, Isabel, Nomakuchi, Tomoki T., Tetenbaum-Novatt, Jaclyn, Rahman, Mohammad Alinoor, Fregoso, Oliver, Rees, Holly, Krainer, Adrian R. (May 2018) Mechanism of Nonsense-Mediated mRNA Decay Stimulation by Splicing Factor SRSF1. Cell Reports, 23 (7). pp. 2186-2198. ISSN 2211-1247

Nomakuchi, T. T., Rigo, F., Aznarez, I., Krainer, A. R. (February 2016) Antisense oligonucleotide-directed inhibition of nonsense-mediated mRNA decay. Nat Biotechnol, 34 (2). pp. 164-166. ISSN 1546-1696 (Electronic)1087-0156 (Linking)

Sahashi, K., Ling, K. K., Hua, Y., Wilkinson, J. E., Nomakuchi, T., Rigo, F., Hung, G., Xu, D., Jiang, Y. P., Lin, R. Z., Ko, C. P., Bennett, C. F., Krainer, A. R. (September 2013) Pathological impact of SMN2 mis-splicing in adult SMA mice. EMBO Molecular Medicine, 5 (10). pp. 1586-1601. ISSN 17574676 (ISSN)

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