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The chemical evolution of oligonucleotide therapies of clinical utility

Nature biotechnology, 2017-03, Vol.35 (3), p.238-248

2017

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Enhancer RNA Facilitates NELF Release from Immediate Early Genes

Molecular cell, 2014-10, Vol.56 (1), p.29-42

2014

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Silencing disease genes in the laboratory and the clinic

The Journal of pathology, 2012-01, Vol.226 (2), p.365-379

2012

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Suppression of mutant C9orf72 expression by a potent mixed backbone antisense oligonucleotide

Nature medicine, 2022-01, Vol.28 (1), p.117-124

2022

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Oligonucleotide therapeutics: chemistry, delivery and clinical progress

Future medicinal chemistry, 2015-10, Vol.7 (16), p.2221-2242

2015

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Heavily and fully modified RNAs guide efficient SpyCas9-mediated genome editing

Nature communications, 2018-07, Vol.9 (1), p.2641-9, Article 2641

2018

Volltextzugriff (PDF)

Genome-wide detection of CRISPR editing in vivo using GUIDE-tag

Nature communications, 2022-01, Vol.13 (1), p.437-437, Article 437

2022

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Domain-inlaid Nme2Cas9 adenine base editors with improved activity and targeting scope

Nature communications, 2024-02, Vol.15 (1), p.1458-1458, Article 1458

2024

Volltextzugriff (PDF)

Quantifying the activity profile of ASO and siRNA conjugates in glioblastoma xenograft tumors in vivo

Nucleic acids research, 2024-05, Vol.52 (9), p.4799

2024

Volltextzugriff (PDF)

Self-delivering, chemically modified CRISPR RNAs for AAV co-delivery and genome editing in vivo

Nucleic acids research, 2024-01, Vol.52 (2), p.977

2024

Volltextzugriff (PDF)

Structure of the catalytically active APOBEC3G bound to a DNA oligonucleotide inhibitor reveals tetrahedral geometry of the transition state

Nature communications, 2022-11, Vol.13 (1), p.7117-10, Article 7117

2022

Volltextzugriff (PDF)

G-rich motifs within phosphorothioate-based antisense oligonucleotides (ASOs) drive activation of FXN expression through indirect effects

Nucleic acids research, 2022-12, Vol.50 (22), p.12657

2022

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Poly(GR) and poly(GA) in cerebrospinal fluid as potential biomarkers for C9ORF72-ALS/FTD

Nature communications, 2022-05, Vol.13 (1), p.2799-2799, Article 2799

2022

Volltextzugriff (PDF)

RNA therapeutics on the rise

Nature reviews. Drug discovery, 2020-07, Vol.19 (7), p.441-442

2020

Volltextzugriff (PDF)

EZH2 inhibition reactivates epigenetically silenced FMR1 and normalizes molecular and electrophysiological abnormalities in fragile X syndrome neurons

Frontiers in neuroscience, 2024-02, Vol.18, p.1348478-1348478

2024

Volltextzugriff (PDF)

Novel Cluster and Monomer-Based GalNAc Structures Induce Effective Uptake of siRNAs in Vitro and in Vivo

Bioconjugate chemistry, 2018-07, Vol.29 (7), p.2478-2488

2018

Volltextzugriff (PDF)

5'-Modifications improve potency and efficacy of DNA donors for precision genome editing

eLife, 2021-10, Vol.10

2021

Volltextzugriff (PDF)

Next-Generation Peptide Nucleic Acid Chimeras Exhibit High Affinity and Potent Gene Silencing

Biochemistry (Easton), 2019-02, Vol.58 (6), p.582-589

2019

Volltextzugriff (PDF)

Dendritic amphiphilic siRNA: Selective albumin binding, in vivo efficacy, and low toxicity

Molecular therapy. Nucleic acids, 2023-12, Vol.34, p.102080-102080, Article 102080

2023

Volltextzugriff (PDF)

Synergistic effects between analogs of DNA and RNA improve the potency of siRNA-mediated gene silencing

Nucleic acids research, 2010-07, Vol.38 (13), p.4547

2010

Volltextzugriff (PDF)

An Investigation into the Potential of Targeting Escherichia coli rne mRNA with Locked Nucleic Acid (LNA) Gapmers as an Antibacterial Strategy

Molecules (Basel, Switzerland), 2021-06, Vol.26 (11), p.3414

2021

Volltextzugriff (PDF)

Single-Stranded Silencing RNAs: Hit Rate and Chemical Modification

Nucleic acid therapeutics, 2016-08, Vol.26 (4), p.216

2016

Volltextzugriff (PDF)

Locked nucleic acid: tighter is different

Chemical communications (Cambridge, England), 2013-06, Vol.49 (50), p.5618-5620

Watts, Jonathan K

2013

Volltextzugriff (PDF)

Studies on the hydrolytic stability of 2'-fluoroarabinonucleic acid (2'F-ANA)

Organic & biomolecular chemistry, 2009-01, Vol.7 (9), p.1904-1910

2009

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Locked Nucleic Acid Gapmers and Conjugates Potently Silence ADAM33, an Asthma-Associated Metalloprotease with Nuclear-Localized mRNA

Molecular therapy. Nucleic acids, 2017-09, Vol.8, p.158-168

2017

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The chemical evolution of oligonucleotide therapies of clinical utility

Enhancer RNA Facilitates NELF Release from Immediate Early Genes

Silencing disease genes in the laboratory and the clinic

Suppression of mutant C9orf72 expression by a potent mixed backbone antisense oligonucleotide

Oligonucleotide therapeutics: chemistry, delivery and clinical progress

Heavily and fully modified RNAs guide efficient SpyCas9-mediated genome editing

Genome-wide detection of CRISPR editing in vivo using GUIDE-tag

Domain-inlaid Nme2Cas9 adenine base editors with improved activity and targeting scope

Quantifying the activity profile of ASO and siRNA conjugates in glioblastoma xenograft tumors in vivo

Self-delivering, chemically modified CRISPR RNAs for AAV co-delivery and genome editing in vivo

Structure of the catalytically active APOBEC3G bound to a DNA oligonucleotide inhibitor reveals tetrahedral geometry of the transition state

G-rich motifs within phosphorothioate-based antisense oligonucleotides (ASOs) drive activation of FXN expression through indirect effects

Poly(GR) and poly(GA) in cerebrospinal fluid as potential biomarkers for C9ORF72-ALS/FTD

RNA therapeutics on the rise

EZH2 inhibition reactivates epigenetically silenced FMR1 and normalizes molecular and electrophysiological abnormalities in fragile X syndrome neurons

Novel Cluster and Monomer-Based GalNAc Structures Induce Effective Uptake of siRNAs in Vitro and in Vivo

5'-Modifications improve potency and efficacy of DNA donors for precision genome editing

Next-Generation Peptide Nucleic Acid Chimeras Exhibit High Affinity and Potent Gene Silencing

Dendritic amphiphilic siRNA: Selective albumin binding, in vivo efficacy, and low toxicity

Synergistic effects between analogs of DNA and RNA improve the potency of siRNA-mediated gene silencing

An Investigation into the Potential of Targeting Escherichia coli rne mRNA with Locked Nucleic Acid (LNA) Gapmers as an Antibacterial Strategy

Single-Stranded Silencing RNAs: Hit Rate and Chemical Modification

Locked nucleic acid: tighter is different

Studies on the hydrolytic stability of 2'-fluoroarabinonucleic acid (2'F-ANA)

Locked Nucleic Acid Gapmers and Conjugates Potently Silence ADAM33, an Asthma-Associated Metalloprotease with Nuclear-Localized mRNA

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