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Single-Stranded RNAs Use RNAi to Potently and Allele-Selectively Inhibit Mutant Huntingtin Expression

Cell, 2012-08, Vol.150 (5), p.895-908

2012

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An Overview of Sugar-Modified Oligonucleotides for Antisense Therapeutics

Chemistry & biodiversity, 2011-09, Vol.8 (9), p.1616-1641

Prakash, Thazha P.

2011

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Overcoming the challenges of tissue delivery for oligonucleotide therapeutics

Trends in pharmacological sciences (Regular ed.), 2021-07, Vol.42 (7), p.588-604

2021

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Single-Stranded siRNAs Activate RNAi in Animals

Cell, 2012-08, Vol.150 (5), p.883-894

2012

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Asialoglycoprotein receptor 1 mediates productive uptake of N-acetylgalactosamine-conjugated and unconjugated phosphorothioate antisense oligonucleotides into liver hepatocytes

Nucleic acids research, 2017-12, Vol.45 (21), p.12388-12400

2017

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Targeted delivery of antisense oligonucleotides to hepatocytes using triantennary N-acetyl galactosamine improves potency 10-fold in mice

Nucleic acids research, 2014-07, Vol.42 (13), p.8796-8807

2014

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Fatty acid conjugation enhances potency of antisense oligonucleotides in muscle

Nucleic acids research, 2019-07, Vol.47 (12), p.6029-6044

2019

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Site-specific incorporation of 5'-methyl DNA enhances the therapeutic profile of gapmer ASOs

Nucleic acids research, 2021-02, Vol.49 (4), p.1828-1839

2021

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Kinetic and subcellular analysis of PS-ASO/protein interactions with P54nrb and RNase H1

Nucleic acids research, 2019-11, Vol.47 (20), p.10865-10880

2019

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Mechanisms of palmitic acid-conjugated antisense oligonucleotide distribution in mice

Nucleic acids research, 2020-05, Vol.48 (8), p.4382-4395

2020

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2'-Modified oligonucleotides for antisense therapeutics

Current topics in medicinal chemistry, 2007-04, Vol.7 (7), p.641-649

2007

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Argonaute 2-dependent Regulation of Gene Expression by Single-stranded miRNA Mimics

Molecular therapy, 2016-05, Vol.24 (5), p.946-955

2016

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Chemically Modified Cpf1-CRISPR RNAs Mediate Efficient Genome Editing in Mammalian Cells

Molecular therapy, 2018-05, Vol.26 (5), p.1228-1240

2018

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Characterizing the effect of GalNAc and phosphorothioate backbone on binding of antisense oligonucleotides to the asialoglycoprotein receptor

Nucleic acids research, 2017-03, Vol.45 (5), p.2294-2306

2017

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Structural Analysis of Human Argonaute‑2 Bound to a Modified siRNA Guide

Journal of the American Chemical Society, 2016-07, Vol.138 (28), p.8694-8697

2016

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Premature transcription termination at the expanded GAA repeats and aberrant alternative polyadenylation contributes to the Frataxin transcriptional deficit in Friedreich's ataxia

Human molecular genetics, 2022-10, Vol.31 (20), p.3539-3557

2022

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Glucagon Like Peptide 1 Receptor Agonists for Targeted Delivery of Antisense Oligonucleotides to Pancreatic Beta Cell

Journal of the American Chemical Society, 2021-03, Vol.143 (9), p.3416-3429

2021

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Enhanced Potency of GalNAc-Conjugated Antisense Oligonucleotides in Hepatocellular Cancer Models

Molecular therapy, 2019-09, Vol.27 (9), p.1547-1557

2019

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Lipid Conjugates Enhance Endosomal Release of Antisense Oligonucleotides Into Cells

Nucleic acid therapeutics, 2019-10, Vol.29 (5), p.245-255

2019

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TCP1 complex proteins interact with phosphorothioate oligonucleotides and can co-localize in oligonucleotide-induced nuclear bodies in mammalian cells

Nucleic acids research, 2014-07, Vol.42 (12), p.7819-7832

2014

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Chop / Ddit3 depletion in β cells alleviates ER stress and corrects hepatic steatosis in mice

Science translational medicine, 2021-07, Vol.13 (604)

2021

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Comprehensive Structure–Activity Relationship of Triantennary N‑Acetylgalactosamine Conjugated Antisense Oligonucleotides for Targeted Delivery to Hepatocytes

Journal of medicinal chemistry, 2016-03, Vol.59 (6), p.2718-2733

2016

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Targeted Delivery of Antisense Oligonucleotides Using Neurotensin Peptides

Journal of medicinal chemistry, 2020-08, Vol.63 (15), p.8471-8484

2020

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Blood Pressure Lowering and Safety Improvements With Liver Angiotensinogen Inhibition in Models of Hypertension and Kidney Injury

Hypertension (Dallas, Tex. 1979), 2017-09, Vol.70 (3), p.566-576

2017

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Conjugation of hydrophobic moieties enhances potency of antisense oligonucleotides in the muscle of rodents and non-human primates

Nucleic acids research, 2019-07, Vol.47 (12), p.6045

2019

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Single-Stranded RNAs Use RNAi to Potently and Allele-Selectively Inhibit Mutant Huntingtin Expression

An Overview of Sugar-Modified Oligonucleotides for Antisense Therapeutics

Overcoming the challenges of tissue delivery for oligonucleotide therapeutics

Single-Stranded siRNAs Activate RNAi in Animals

Asialoglycoprotein receptor 1 mediates productive uptake of N-acetylgalactosamine-conjugated and unconjugated phosphorothioate antisense oligonucleotides into liver hepatocytes

Targeted delivery of antisense oligonucleotides to hepatocytes using triantennary N-acetyl galactosamine improves potency 10-fold in mice

Fatty acid conjugation enhances potency of antisense oligonucleotides in muscle

Site-specific incorporation of 5'-methyl DNA enhances the therapeutic profile of gapmer ASOs

Kinetic and subcellular analysis of PS-ASO/protein interactions with P54nrb and RNase H1

Mechanisms of palmitic acid-conjugated antisense oligonucleotide distribution in mice

2'-Modified oligonucleotides for antisense therapeutics

Argonaute 2-dependent Regulation of Gene Expression by Single-stranded miRNA Mimics

Chemically Modified Cpf1-CRISPR RNAs Mediate Efficient Genome Editing in Mammalian Cells

Characterizing the effect of GalNAc and phosphorothioate backbone on binding of antisense oligonucleotides to the asialoglycoprotein receptor

Structural Analysis of Human Argonaute‑2 Bound to a Modified siRNA Guide

Premature transcription termination at the expanded GAA repeats and aberrant alternative polyadenylation contributes to the Frataxin transcriptional deficit in Friedreich's ataxia

Glucagon Like Peptide 1 Receptor Agonists for Targeted Delivery of Antisense Oligonucleotides to Pancreatic Beta Cell

Enhanced Potency of GalNAc-Conjugated Antisense Oligonucleotides in Hepatocellular Cancer Models

Lipid Conjugates Enhance Endosomal Release of Antisense Oligonucleotides Into Cells

TCP1 complex proteins interact with phosphorothioate oligonucleotides and can co-localize in oligonucleotide-induced nuclear bodies in mammalian cells

Chop / Ddit3 depletion in β cells alleviates ER stress and corrects hepatic steatosis in mice

Comprehensive Structure–Activity Relationship of Triantennary N‑Acetylgalactosamine Conjugated Antisense Oligonucleotides for Targeted Delivery to Hepatocytes

Targeted Delivery of Antisense Oligonucleotides Using Neurotensin Peptides

Blood Pressure Lowering and Safety Improvements With Liver Angiotensinogen Inhibition in Models of Hypertension and Kidney Injury

Conjugation of hydrophobic moieties enhances potency of antisense oligonucleotides in the muscle of rodents and non-human primates

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