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2'-O-methoxyethyl splice-switching oligos correct splicing from IVS2-745 β-thalassemia patient cells restoring HbA production and chain rebalance.
Dong A, Ghiaccio V, Motta I, Guo S, Peralta R, Freier SM, Watt A, Damle S, Ikawa Y, Jarocha D, Chappell M, Stephanou C, Delbini P, Chen C, Christou S, Kleanthous M, Smith-Whitley K, Manwani D, Casu C, Abdulmalik O, Cappellini MD, Rivella S, Breda L. Dong A, et al. Haematologica. 2019 May 1;106(5):1433-1442. doi: 10.3324/haematol.2019.226852. Haematologica. 2019. PMID: 32439726 Free PMC article.
Phospholamban antisense oligonucleotides improve cardiac function in murine cardiomyopathy.
Grote Beverborg N, Später D, Knöll R, Hidalgo A, Yeh ST, Elbeck Z, Silljé HHW, Eijgenraam TR, Siga H, Zurek M, Palmér M, Pehrsson S, Albery T, Bomer N, Hoes MF, Boogerd CJ, Frisk M, van Rooij E, Damle S, Louch WE, Wang QD, Fritsche-Danielson R, Chien KR, Hansson KM, Mullick AE, de Boer RA, van der Meer P. Grote Beverborg N, et al. Nat Commun. 2021 Aug 30;12(1):5180. doi: 10.1038/s41467-021-25439-0. Nat Commun. 2021. PMID: 34462437 Free PMC article.
36 results