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TricycloDNA-modified oligo-2′-deoxyribonucleotides reduce scavenger receptor B1 mRNA in hepatic and extra-hepatic tissues—a comparative study of oligonucleotide length, design and chemistry

Nucleic acids research, 2012-07, Vol.40 (13), p.6135-6143

2012

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Selective tissue targeting of synthetic nucleic acid drugs

The Journal of clinical investigation, 2019-03, Vol.129 (3), p.915-925

2019

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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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Towards next generation antisense oligonucleotides: mesylphosphoramidate modification improves therapeutic index and duration of effect of gapmer antisense oligonucleotides

Nucleic acids research, 2021-09, Vol.49 (16), p.9026-9041

2021

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Chemical modification of PS-ASO therapeutics reduces cellular protein-binding and improves the therapeutic index

Nature biotechnology, 2019-06, Vol.37 (6), p.640-650

2019

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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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Single-Stranded siRNAs Activate RNAi in Animals

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

2012

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Origins of the Increased Affinity of Phosphorothioate-Modified Therapeutic Nucleic Acids for Proteins

Journal of the American Chemical Society, 2020-04, Vol.142 (16), p.7456-7468

2020

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Stabilin-1 and Stabilin-2 are specific receptors for the cellular internalization of phosphorothioate-modified antisense oligonucleotides (ASOs) in the liver

Nucleic acids research, 2016-04, Vol.44 (6), p.2782-2794

2016

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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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Understanding the effect of controlling phosphorothioate chirality in the DNA gap on the potency and safety of gapmer antisense oligonucleotides

Nucleic acids research, 2020-02, Vol.48 (4), p.1691-1700

2020

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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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Hepatotoxicity of high affinity gapmer antisense oligonucleotides is mediated by RNase H1 dependent promiscuous reduction of very long pre-mRNA transcripts

Nucleic acids research, 2016-03, Vol.44 (5), p.2093-2109

2016

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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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The Medicinal Chemistry of Therapeutic Oligonucleotides

Journal of medicinal chemistry, 2016-11, Vol.59 (21), p.9645-9667

2016

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DNA/RNA heteroduplex oligonucleotide for highly efficient gene silencing

Nature communications, 2015-08, Vol.6 (1), p.7969, Article 7969

2015

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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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Rational design of antisense oligonucleotides targeting single nucleotide polymorphisms for potent and allele selective suppression of mutant Huntingtin in the CNS

Nucleic acids research, 2013-11, Vol.41 (21), p.9634-9650

2013

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Synthesis, biophysical properties and biological activity of second generation antisense oligonucleotides containing chiral phosphorothioate linkages

Nucleic acids research, 2014-12, Vol.42 (22), p.13456-13468

2014

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Simultaneous inhibition of endocytic recycling and lysosomal fusion sensitizes cells and tissues to oligonucleotide therapeutics

Nucleic acids research, 2023-02, Vol.51 (4), p.1583-1599

2023

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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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In Vivo Evaluation of Candidate Allele-specific Mutant Huntingtin Gene Silencing Antisense Oligonucleotides

Molecular therapy, 2014-12, Vol.22 (12), p.2093-2106

2014

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Conception and Synthesis of Oxabicyclic Nucleoside Phosphonates as Internucleotidic Phosphate Surrogates in Antisense Oligonucleotide Constructs

Organic letters, 2018-09, Vol.20 (17), p.5296-5299

2018

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Allele-specific suppression of mutant huntingtin using antisense oligonucleotides: providing a therapeutic option for all Huntington disease patients

PloS one, 2014-09, Vol.9 (9), p.e107434-e107434

2014

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TricycloDNA-modified oligo-2′-deoxyribonucleotides reduce scavenger receptor B1 mRNA in hepatic and extra-hepatic tissues—a comparative study of oligonucleotide length, design and chemistry

Selective tissue targeting of synthetic nucleic acid drugs

Overcoming the challenges of tissue delivery for oligonucleotide therapeutics

Towards next generation antisense oligonucleotides: mesylphosphoramidate modification improves therapeutic index and duration of effect of gapmer antisense oligonucleotides

Chemical modification of PS-ASO therapeutics reduces cellular protein-binding and improves the therapeutic index

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

Single-Stranded siRNAs Activate RNAi in Animals

Origins of the Increased Affinity of Phosphorothioate-Modified Therapeutic Nucleic Acids for Proteins

Stabilin-1 and Stabilin-2 are specific receptors for the cellular internalization of phosphorothioate-modified antisense oligonucleotides (ASOs) in the liver

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

Understanding the effect of controlling phosphorothioate chirality in the DNA gap on the potency and safety of gapmer antisense oligonucleotides

Mechanisms of palmitic acid-conjugated antisense oligonucleotide distribution in mice

Hepatotoxicity of high affinity gapmer antisense oligonucleotides is mediated by RNase H1 dependent promiscuous reduction of very long pre-mRNA transcripts

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

The Medicinal Chemistry of Therapeutic Oligonucleotides

DNA/RNA heteroduplex oligonucleotide for highly efficient gene silencing

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

Rational design of antisense oligonucleotides targeting single nucleotide polymorphisms for potent and allele selective suppression of mutant Huntingtin in the CNS

Synthesis, biophysical properties and biological activity of second generation antisense oligonucleotides containing chiral phosphorothioate linkages

Simultaneous inhibition of endocytic recycling and lysosomal fusion sensitizes cells and tissues to oligonucleotide therapeutics

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

In Vivo Evaluation of Candidate Allele-specific Mutant Huntingtin Gene Silencing Antisense Oligonucleotides

Conception and Synthesis of Oxabicyclic Nucleoside Phosphonates as Internucleotidic Phosphate Surrogates in Antisense Oligonucleotide Constructs

Allele-specific suppression of mutant huntingtin using antisense oligonucleotides: providing a therapeutic option for all Huntington disease patients

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