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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.
Rational design of antisense oligonucleotides targeting single nucleotide polymorphisms for potent and allele selective suppression of mutant Huntingtin in the CNS.
Østergaard ME, Southwell AL, Kordasiewicz H, Watt AT, Skotte NH, Doty CN, Vaid K, Villanueva EB, Swayze EE, Bennett CF, Hayden MR, Seth PP. Østergaard ME, et al. Nucleic Acids Res. 2013 Nov;41(21):9634-50. doi: 10.1093/nar/gkt725. Epub 2013 Aug 19. Nucleic Acids Res. 2013. PMID: 23963702 Free PMC article.
Allele-specific suppression of mutant huntingtin using antisense oligonucleotides: providing a therapeutic option for all Huntington disease patients.
Skotte NH, Southwell AL, Østergaard ME, Carroll JB, Warby SC, Doty CN, Petoukhov E, Vaid K, Kordasiewicz H, Watt AT, Freier SM, Hung G, Seth PP, Bennett CF, Swayze EE, Hayden MR. Skotte NH, et al. PLoS One. 2014 Sep 10;9(9):e107434. doi: 10.1371/journal.pone.0107434. eCollection 2014. PLoS One. 2014. PMID: 25207939 Free PMC article.
646 results