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Ligand conjugated antisense oligonucleotide for the treatment of transthyretin amyloidosis: preclinical and phase 1 data.
Viney NJ, Guo S, Tai LJ, Baker BF, Aghajan M, Jung SW, Yu RZ, Booten S, Murray H, Machemer T, Burel S, Murray S, Buchele G, Tsimikas S, Schneider E, Geary RS, Benson MD, Monia BP. Viney NJ, et al. Among authors: booten s. ESC Heart Fail. 2021 Feb;8(1):652-661. doi: 10.1002/ehf2.13154. Epub 2020 Dec 7. ESC Heart Fail. 2021. PMID: 33283485 Free PMC article. Clinical Trial.
Targeted delivery of antisense oligonucleotides to hepatocytes using triantennary N-acetyl galactosamine improves potency 10-fold in mice.
Prakash TP, Graham MJ, Yu J, Carty R, Low A, Chappell A, Schmidt K, Zhao C, Aghajan M, Murray HF, Riney S, Booten SL, Murray SF, Gaus H, Crosby J, Lima WF, Guo S, Monia BP, Swayze EE, Seth PP. Prakash TP, et al. Among authors: booten sl. Nucleic Acids Res. 2014 Jul;42(13):8796-807. doi: 10.1093/nar/gku531. Epub 2014 Jul 3. Nucleic Acids Res. 2014. PMID: 24992960 Free PMC article.
Comprehensive Structure-Activity Relationship of Triantennary N-Acetylgalactosamine Conjugated Antisense Oligonucleotides for Targeted Delivery to Hepatocytes.
Prakash TP, Yu J, Migawa MT, Kinberger GA, Wan WB, Østergaard ME, Carty RL, Vasquez G, Low A, Chappell A, Schmidt K, Aghajan M, Crosby J, Murray HM, Booten SL, Hsiao J, Soriano A, Machemer T, Cauntay P, Burel SA, Murray SF, Gaus H, Graham MJ, Swayze EE, Seth PP. Prakash TP, et al. J Med Chem. 2016 Mar 24;59(6):2718-33. doi: 10.1021/acs.jmedchem.5b01948. Epub 2016 Mar 9. J Med Chem. 2016. PMID: 26914862
Antisense oligonucleotide treatment ameliorates IFN-γ-induced proteinuria in APOL1-transgenic mice.
Aghajan M, Booten SL, Althage M, Hart CE, Ericsson A, Maxvall I, Ochaba J, Menschik-Lundin A, Hartleib J, Kuntz S, Gattis D, Ahlström C, Watt AT, Engelhardt JA, Monia BP, Magnone MC, Guo S. Aghajan M, et al. Among authors: booten sl. JCI Insight. 2019 Jun 20;4(12):e126124. doi: 10.1172/jci.insight.126124. eCollection 2019 Jun 20. JCI Insight. 2019. PMID: 31217349 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: booten s. 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.
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