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Chemical modification of PS-ASO therapeutics reduces cellular protein-binding and improves the therapeutic index.
Shen W, De Hoyos CL, Migawa MT, Vickers TA, Sun H, Low A, Bell TA 3rd, Rahdar M, Mukhopadhyay S, Hart CE, Bell M, Riney S, Murray SF, Greenlee S, Crooke RM, Liang XH, Seth PP, Crooke ST. Shen W, et al. Among authors: hart ce. Nat Biotechnol. 2019 Jun;37(6):640-650. doi: 10.1038/s41587-019-0106-2. Epub 2019 Apr 29. Nat Biotechnol. 2019. PMID: 31036929
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: hart ce. 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 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: hart ce. 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.
147 results