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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. Among authors: watt a. Haematologica. 2019 May 1;106(5):1433-1442. doi: 10.3324/haematol.2019.226852. Haematologica. 2019. PMID: 32439726 Free PMC article.
Reducing TMPRSS6 ameliorates hemochromatosis and β-thalassemia in mice.
Guo S, Casu C, Gardenghi S, Booten S, Aghajan M, Peralta R, Watt A, Freier S, Monia BP, Rivella S. Guo S, et al. Among authors: watt a. J Clin Invest. 2013 Apr;123(4):1531-41. doi: 10.1172/JCI66969. Epub 2013 Mar 25. J Clin Invest. 2013. PMID: 23524968 Free PMC article.
Hepatotoxicity of high affinity gapmer antisense oligonucleotides is mediated by RNase H1 dependent promiscuous reduction of very long pre-mRNA transcripts.
Burel SA, Hart CE, Cauntay P, Hsiao J, Machemer T, Katz M, Watt A, Bui HH, Younis H, Sabripour M, Freier SM, Hung G, Dan A, Prakash TP, Seth PP, Swayze EE, Bennett CF, Crooke ST, Henry SP. Burel SA, et al. Among authors: watt a. Nucleic Acids Res. 2016 Mar 18;44(5):2093-109. doi: 10.1093/nar/gkv1210. Epub 2015 Nov 8. Nucleic Acids Res. 2016. PMID: 26553810 Free PMC article.
Antisense oligonucleotides to therapeutically target SARS-CoV-2 infection.
Qiao Y, Wotring JW, Zhang CJ, Jiang X, Xiao L, Watt A, Gattis D, Scandalis E, Freier S, Zheng Y, Pretto CD, Ellison SJ, Swayze EE, Guo S, Sexton JZ, Chinnaiyan AM. Qiao Y, et al. Among authors: watt a. PLoS One. 2023 Feb 3;18(2):e0281281. doi: 10.1371/journal.pone.0281281. eCollection 2023. PLoS One. 2023. PMID: 36735698 Free PMC article.
646 results