Aartsma-Rus, A., & Krieg, A. M. (2017). FDA Approves Eteplirsen for
Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga.Nucleic Acid Therapeutics, 27(1), 1–3.
https://doi.org/10.1089/nat.2016.0657
Bilandzic, M., Rainczuk, A., Green, E., Fairweather, N., Jobling, T. W.,
Plebanski, M., & Stephens, A. N. (2019). Keratin-14 (KRT14) Positive
Leader Cells Mediate Mesothelial Clearance and Invasion by Ovarian
Cancer Cells. Cancers, 11(9).
https://doi.org/10.3390/cancers11091228
Cartegni, L., Wang, J., Zhu, Z., Zhang, M. Q., & Krainer, A. R. (2003).
ESEfinder: A web resource to identify exonic splicing enhancers.Nucleic Acids Research, 31(13), 3568–3571.
Cong, L., Ran, F. A., Cox, D., Lin, S., Barretto, R., Habib, N., Hsu, P.
D., Wu, X., Jiang, W., Marraffini, L. A., & Zhang, F. (2013). Multiplex
Genome Engineering Using CRISPR/Cas Systems. Science,339(6121), 819–823. https://doi.org/10.1126/science.1231143
Ekker, S. C. (2006). Morphants: A New Systematic Vertebrate Functional
Genomics Approach. Yeast (Chichester, England), 17(4),
302–306.
https://doi.org/10.1002/1097-0061(200012)17:4<302::AID-YEA53>3.0.CO;2-#
Geissler, R., Simkin, A., Floss, D., Patel, R., Fogarty, E. A.,
Scheller, J., & Grimson, A. (2016). A widespread sequence-specific mRNA
decay pathway mediated by hnRNPs A1 and A2/B1. Genes &
Development, 30(9), 1070–1085.
https://doi.org/10.1101/gad.277392.116
Gilbert, L. A., Horlbeck, M. A., Adamson, B., Villalta, J. E., Chen, Y.,
Whitehead, E. H., Guimaraes, C., Panning, B., Ploegh, H. L., Bassik, M.
C., Qi, L. S., Kampmann, M., & Weissman, J. S. (2014). Genome-Scale
CRISPR-Mediated Control of Gene Repression and Activation. Cell,159(3), 647–661. https://doi.org/10.1016/j.cell.2014.09.029
Jain, N., Lin, H.-C., Morgan, C. E., Harris, M. E., & Tolbert, B. S.
(2017). Rules of RNA specificity of hnRNP A1 revealed by global and
quantitative analysis of its affinity distribution. Proceedings of
the National Academy of Sciences of the United States of America,114(9), 2206–2211. https://doi.org/10.1073/pnas.1616371114
Jean-Philippe, J., Paz, S., & Caputi, M. (2013). hnRNP A1: The Swiss
Army Knife of Gene Expression. International Journal of Molecular
Sciences, 14(9), 18999–19024.
https://doi.org/10.3390/ijms140918999
Kampmann, M. (2018). CRISPRi and CRISPRa Screens in Mammalian Cells for
Precision Biology and Medicine. ACS Chemical Biology,13(2), 406–416. https://doi.org/10.1021/acschembio.7b00657
Kashima, T., Rao, N., David, C. J., & Manley, J. L. (2007). HnRNP A1
functions with specificity in repression of SMN2 exon 7 splicing.Human Molecular Genetics, 16(24), 3149–3159.
https://doi.org/10.1093/hmg/ddm276
Koike-Yusa, H., Li, Y., Tan, E.-P., Velasco-Herrera, M. D. C., & Yusa,
K. (2014). Genome-wide recessive genetic screening in mammalian cells
with a lentiviral CRISPR-guide RNA library. Nature Biotechnology,32(3), 267–273. https://doi.org/10.1038/nbt.2800
Lee, Y., & Rio, D. C. (2015). Mechanisms and Regulation of Alternative
Pre-mRNA Splicing. Annual Review of Biochemistry, 84,
291–323. https://doi.org/10.1146/annurev-biochem-060614-034316
Mullenders, J., Fabius, A. W. M., Madiredjo, M., Bernards, R., &
Beijersbergen, R. L. (2009). A Large Scale shRNA Barcode Screen
Identifies the Circadian Clock Component ARNTL as Putative Regulator of
the p53 Tumor Suppressor Pathway. PLoS ONE, 4(3).
https://doi.org/10.1371/journal.pone.0004798
Nogrady, B. (2019). The challenge of delivering RNA-interference
therapeutics to their target cells. Nature, 574(7778),
S8–S9. https://doi.org/10.1038/d41586-019-03071-9
Papafotiou, G., Paraskevopoulou, V., Vasilaki, E., Kanaki, Z.,
Paschalidis, N., & Klinakis, A. (2016). KRT14 marks a subpopulation of
bladder basal cells with pivotal role in regeneration and tumorigenesis.Nature Communications, 7(1), 11914.
https://doi.org/10.1038/ncomms11914
Roberts, T. C., Langer, R., & Wood, M. J. A. (2020). Advances in
oligonucleotide drug delivery. Nature Reviews Drug Discovery,19(10), 673–694. https://doi.org/10.1038/s41573-020-0075-7
Stein, C. A., & Castanotto, D. (2017). FDA-Approved Oligonucleotide
Therapies in 2017. Molecular Therapy, 25(5), 1069–1075.
https://doi.org/10.1016/j.ymthe.2017.03.023
Tycko, J., Wainberg, M., Marinov, G. K., Ursu, O., Hess, G. T., Ego, B.
K., Aradhana, Li, A., Truong, A., Trevino, A. E., Spees, K., Yao, D.,
Kaplow, I. M., Greenside, P. G., Morgens, D. W., Phanstiel, D. H.,
Snyder, M. P., Bintu, L., Greenleaf, W. J., … Bassik, M. C.
(2019). Mitigation of off-target toxicity in CRISPR-Cas9 screens for
essential non-coding elements. Nature Communications,10(1), 4063. https://doi.org/10.1038/s41467-019-11955-7
Zhu, L.-F., Hu, Y., Yang, C.-C., Xu, X.-H., Ning, T.-Y., Wang, Z.-L.,
Ye, J.-H., & Liu, L.-K. (2012). Snail overexpression induces an
epithelial to mesenchymal transition and cancer stem cell-like
properties in SCC9 cells. Laboratory Investigation, 92(5),
744–752. https://doi.org/10.1038/labinvest.2012.8