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Jbara A, Lin KT, Stossel C, et al. (2023) RBFOX2 modulates a metastatic signature of alternative splicing in pancreatic cancer. Nature
Hastings ML, Krainer AR. (2023) RNA therapeutics. Rna (New York, N.Y.). 29: 393-395
Kim YJ, Nomakuchi T, Papaleonidopoulou F, et al. (2022) Gene-specific nonsense-mediated mRNA decay targeting for cystic fibrosis therapy. Nature Communications. 13: 2978
Gao Y, Lin KT, Jiang T, et al. (2022) Systematic characterization of short intronic splicing-regulatory elements in SMN2 pre-mRNA. Nucleic Acids Research
Kim YJ, Sivetz N, Layne J, et al. (2022) Exon-skipping antisense oligonucleotides for cystic fibrosis therapy. Proceedings of the National Academy of Sciences of the United States of America. 119
Ajiro M, Awaya T, Kim YJ, et al. (2021) Therapeutic manipulation of IKBKAP mis-splicing with a small molecule to cure familial dysautonomia. Nature Communications. 12: 4507
Frederiksen SB, Holm LL, Larsen MR, et al. (2020) Identification of SRSF10 as a regulator of SMN2 ISS-N1. Human Mutation
Sheng L, Rigo F, Bennett CF, et al. (2020) Comparison of the efficacy of MOE and PMO modifications of systemic antisense oligonucleotides in a severe SMA mouse model. Nucleic Acids Research
Rahman MA, Lin KT, Bradley RK, et al. (2020) Recurrent SRSF2 mutations in MDS affect both splicing and NMD. Genes & Development
Rahman MA, Krainer AR, Abdel-Wahab O. (2020) SnapShot: Splicing Alterations in Cancer. Cell. 180: 208-208.e1