Chemical modification of PS-ASO therapeutics reduces cellular protein-binding and improves the therapeutic index
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Christopher E. Hart | S. Mukhopadhyay | S. Crooke | S. Murray | R. Crooke | T. Vickers | Audrey Low | Stan J. Riney | P. Seth | Sarah Greenlee | Meghdad Rahdar | Wen Shen | Xue-hai Liang | M. Migawa | Hong Sun | T. Bell | C. L. De Hoyos | Melanie Bell | S. Crooke
[1] KasuyaTakeshi,et al. Role of Computationally Evaluated Target Specificity in the Hepatotoxicity of Gapmer Antisense Oligonucleotides. , 2018 .
[2] T. Abe,et al. Loss of Sfpq Causes Long-Gene Transcriptopathy in the Brain. , 2018, Cell reports.
[3] S. Crooke,et al. Cellular uptake mediated by epidermal growth factor receptor facilitates the intracellular activity of phosphorothioate-modified antisense oligonucleotides , 2018, Nucleic acids research.
[4] S. Crooke,et al. Acute hepatotoxicity of 2′ fluoro-modified 5–10–5 gapmer phosphorothioate oligonucleotides in mice correlates with intracellular protein binding and the loss of DBHS proteins , 2018, Nucleic acids research.
[5] Howard Y. Chang,et al. Tissue-selective effects of nucleolar stress and rDNA damage in developmental disorders , 2018, Nature.
[6] T. Singer,et al. A Sensitive In Vitro Approach to Assess the Hybridization-Dependent Toxic Potential of High Affinity Gapmer Oligonucleotides , 2017, Molecular therapy. Nucleic acids.
[7] S. Crooke,et al. Nucleic acid binding proteins affect the subcellular distribution of phosphorothioate antisense oligonucleotides , 2017, Nucleic acids research.
[8] S. Crooke,et al. Dynamic nucleoplasmic and nucleolar localization of mammalian RNase H1 in response to RNAP I transcriptional R-loops , 2017, Nucleic acids research.
[9] T. Gant,et al. Strategies for In Vivo Screening and Mitigation of Hepatotoxicity Associated with Antisense Drugs , 2017, Molecular therapy. Nucleic acids.
[10] S. Crooke,et al. RNase H1-Dependent Antisense Oligonucleotides Are Robustly Active in Directing RNA Cleavage in Both the Cytoplasm and the Nucleus , 2017, Molecular therapy : the journal of the American Society of Gene Therapy.
[11] H. Bohr,et al. Electronic Structures of LNA Phosphorothioate Oligonucleotides , 2017, Molecular therapy. Nucleic acids.
[12] T. CrookeStanley. Molecular Mechanisms of Antisense Oligonucleotides. , 2017 .
[13] S. Crooke,et al. Depletion of NEAT1 lncRNA attenuates nucleolar stress by releasing sequestered P54nrb and PSF to facilitate c-Myc translation , 2017, PloS one.
[14] Stanley T Crooke,et al. Cellular uptake and trafficking of antisense oligonucleotides , 2017, Nature Biotechnology.
[15] anastasia. khvorova,et al. The chemical evolution of oligonucleotide therapies of clinical utility , 2017, Nature Biotechnology.
[16] S. Crooke,et al. Development of a Quantitative BRET Affinity Assay for Nucleic Acid-Protein Interactions , 2016, PloS one.
[17] T. Kasuya,et al. Ribonuclease H1-dependent hepatotoxicity caused by locked nucleic acid-modified gapmer antisense oligonucleotides , 2016, Scientific Reports.
[18] T. Singer,et al. Establishment of a Predictive In Vitro Assay for Assessment of the Hepatotoxic Potential of Oligonucleotide Drugs , 2016, PloS one.
[19] Christopher E. Hart,et al. Viable RNaseH1 knockout mice show RNaseH1 is essential for R loop processing, mitochondrial and liver function , 2016, Nucleic acids research.
[20] A. Ryan,et al. Development of a Method for Profiling Protein Interactions with LNA-Modified Antisense Oligonucleotides Using Protein Microarrays. , 2016, Nucleic acid therapeutics.
[21] S. Crooke,et al. Hsp90 protein interacts with phosphorothioate oligonucleotides containing hydrophobic 2′-modifications and enhances antisense activity , 2016, Nucleic acids research.
[22] Christopher E. Hart,et al. Hepatotoxicity of high affinity gapmer antisense oligonucleotides is mediated by RNase H1 dependent promiscuous reduction of very long pre-mRNA transcripts , 2015, Nucleic acids research.
[23] Steven A. Brown,et al. Mutations in NONO lead to syndromic intellectual disability and inhibitory synaptic defects , 2015, Nature Neuroscience.
[24] Emma L. Koppe,et al. In silico and in vitro evaluation of exonic and intronic off-target effects form a critical element of therapeutic ASO gapmer optimization , 2015, Nucleic acids research.
[25] S. Crooke,et al. The rates of the major steps in the molecular mechanism of RNase H1-dependent antisense oligonucleotide induced degradation of RNA , 2015, Nucleic acids research.
[26] T. P. Prakash,et al. Efficient Synthesis and Biological Evaluation of 5'-GalNAc Conjugated Antisense Oligonucleotides. , 2015, Bioconjugate chemistry.
[27] J. Lieberman,et al. Apoptosis Triggers Specific, Rapid, and Global mRNA Decay with 3′ Uridylated Intermediates Degraded by DIS3L2 , 2015, Cell reports.
[28] S. Crooke,et al. 2′-Fluoro-modified phosphorothioate oligonucleotide can cause rapid degradation of P54nrb and PSF , 2015, Nucleic acids research.
[29] S. Crooke,et al. Identification and characterization of intracellular proteins that bind oligonucleotides with phosphorothioate linkages , 2022 .
[30] Kendall S. Frazier,et al. Antisense Oligonucleotide Therapies , 2015, Toxicologic pathology.
[31] S. Crooke,et al. Phosphorothioate oligonucleotides can displace NEAT1 RNA and form nuclear paraspeckle-like structures , 2014, Nucleic acids research.
[32] S. Crooke,et al. TCP1 complex proteins interact with phosphorothioate oligonucleotides and can co-localize in oligonucleotide-induced nuclear bodies in mammalian cells , 2014, Nucleic acids research.
[33] H. Bohr,et al. Quantum mechanical studies of DNA and LNA. , 2014, Nucleic acid therapeutics.
[34] A. Burdick,et al. Comparison of hepatic transcription profiles of locked ribonucleic acid antisense oligonucleotides: evidence of distinct pathways contributing to non-target mediated toxicity in mice. , 2014, Toxicological sciences : an official journal of the Society of Toxicology.
[35] A. Burdick,et al. Sequence motifs associated with hepatotoxicity of locked nucleic acid—modified antisense oligonucleotides , 2014, Nucleic acids research.
[36] M. Lindow,et al. Hepatotoxic potential of therapeutic oligonucleotides can be predicted from their sequence and modification pattern. , 2013, Nucleic acid therapeutics.
[37] Stormy J. Chamberlain,et al. Topoisomerases facilitate transcription of long genes linked to autism , 2013, Nature.
[38] S. Crooke,et al. Human RNase H1 Is Associated with Protein P32 and Is Involved in Mitochondrial Pre-rRNA Processing , 2013, PloS one.
[39] J. Kreeger,et al. Chemical modification study of antisense gapmers. , 2012, Nucleic acid therapeutics.
[40] Stephanie C Huelga,et al. Divergent roles of ALS-linked proteins FUS/TLS and TDP-43 intersect in processing long pre-mRNAs , 2012, Nature Neuroscience.
[41] Gene W. Yeo,et al. Long pre-mRNA depletion and RNA missplicing contribute to neuronal vulnerability from loss of TDP-43 , 2011, Nature Neuroscience.
[42] E. Swayze,et al. The Medicinal Chemistry of Oligonucleotides , 2008 .
[43] B. Monia,et al. Antisense oligonucleotides containing locked nucleic acid improve potency but cause significant hepatotoxicity in animals , 2006, Nucleic acids research.
[44] S. Crooke,et al. Determination of the Role of the Human RNase H1 in the Pharmacology of DNA-like Antisense Drugs* , 2004, Journal of Biological Chemistry.
[45] Y. Shav-Tal,et al. PSF and p54nrb/NonO – multi‐functional nuclear proteins , 2002, FEBS letters.
[46] D. Spector,et al. Phosphorothioate antisense oligonucleotides induce the formation of nuclear bodies. , 1998, Molecular biology of the cell.
[47] M. Nerenberg,et al. Effect of phosphorothioate modification of oligodeoxynucleotides on specific protein binding. , 1994, The Journal of biological chemistry.
[48] Y. Cheng,et al. Phosphorothioate oligonucleotides are inhibitors of human DNA polymerases and RNase H: implications for antisense technology. , 1992, Molecular pharmacology.
[49] D. Schlessinger,et al. Differences in rRNA metabolism of primary and SV40-transformed human fibroblasts , 1978, Cell.