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Gomez Limia, Cintia; Baird, Megan; Schwartz, Maura; Saxena, Smita; Meyer, Kathrin; Wein, Nicolas (2022). Emerging Perspectives on Gene Therapy Delivery for Neurodegenerative and Neuromuscular Disorders. Journal of personalized medicine, 12(12) MDPI 10.3390/jpm12121979
Pilotto, Federica; Schmitz, Alexander; Maharjan, Niran; Diab, Rim; Odriozola, Adolfo; Tripathi, Priyanka; Yamoah, Alfred; Scheidegger, Olivier; Oestmann, Angelina; Dennys, Cassandra N; Sinha Ray, Shrestha; Rodrigo, Rochelle; Kolb, Stephen; Aronica, Eleonora; Di Santo, Stefano; Widmer, Hans Rudolf; Charlet-Berguerand, Nicolas; Selvaraj, Bhuvaneish T; Chandran, Siddharthan; Meyer, Kathrin; ... (2022). PolyGA targets the ER stress-adaptive response by impairing GRP75 function at the MAM in C9ORF72-ALS/FTD. Acta neuropathologica, 144(5), pp. 939-966. Springer-Verlag 10.1007/s00401-022-02494-5
Duque, Sandra I; Arnold, W David; Odermatt, Philipp; Li, Xiaohui; Porensky, Paul N; Schmelzer, Leah; Meyer, Kathrin; Kolb, Stephen J; Schümperli, Daniel; Kaspar, Brian K; Burghes, Arthur H M (2015). A large animal model of spinal muscular atrophy and correction of phenotype. Annals of neurology, 77(3), pp. 399-414. Wiley-Blackwell 10.1002/ana.24332
Huo, Qing; Kayikci, Melis; Odermatt, Philipp; Meyer, Kathrin; Michels, Olivia; Saxena, Smita; Ule, Jernej; Schümperli, Daniel (2014). Splicing changes in SMA mouse motoneurons and SMN-depleted neuroblastoma cells: evidence for involvement of splicing regulatory proteins. RNA biology, 11(11), pp. 1430-1446. Landes Bioscience 10.1080/15476286.2014.996494
Voigt, Tilman; Meyer, Kathrin; Baum, Oliver; Schümperli, Daniel (2010). Ultrastructural changes in diaphragm neuromuscular junctions in a severe mouse model for Spinal Muscular Atrophy and their prevention by bifunctional U7 snRNA correcting SMN2 splicing. Neuromuscular disorders, 20(11), pp. 744-52. Amsterdam: Elsevier 10.1016/j.nmd.2010.06.010
Nlend, Rachel Nlend; Meyer, Kathrin; Schümperli, Daniel (2010). Repair of pre-mRNA splicing: Prospects for a therapy for Spinal Muscular Atrophy. RNA biology, 7(4), pp. 730-740. Georgetown, Tex.: Landes Bioscience 10.4161/rna.7.4.12206
Meyer, Kathrin; Marquis, Julien; Trüb, Judith; Nlend Nlend, Rachel; Verp, Sonia; Ruepp, Marc-David; Imboden, Hans; Barde, Isabelle; Trono, Didier; Schümperli, Daniel (2009). Rescue of a severe mouse model for spinal muscular atrophy by U7 snRNA-mediated splicing modulation. Human molecular genetics, 18(3), pp. 546-555. Oxford: Oxford University Press 10.1093/hmg/ddn382
Marquis, Julien; Meyer, Kathrin; Angehrn, Larissa; Kämpfer, Sacha; Rothen-Rutishauser, Barbara; Schümperli, Daniel (2007). Spinal muscular atrophy: SMN2 pre-mRNA splicing corrected by a U7 snRNA derivative carrying a splicing enhancer sequence. Molecular therapy, 15(8), pp. 1479-1486. New York, N.Y.: Nature Publishing Group 10.1038/sj.mt.6300200
Scholl, Raphael; Marquis, Julien; Meyer, Kathrin; Schümperli, Daniel (2007). Spinal Muscular Atrophy: position and functional importance of the branch site preceding SMN exon 7. RNA biology, 4(1), pp. 34-37. Georgetown, Tex.: Landes Bioscience 10.4161/rna.4.1.4534
