Mesyl Phosphoramidate Oligonucleotides as Potential Splice-Switching Agents: Impact of Backbone Structure on Activity and Intracellular Localization

Golodirsen: First Approval

(2020) Young-A Heo   DRUGS

Comparison of the efficacy of MOE and PMO modifications of systemic antisense oligonucleotides in a severe SMA mouse model

(2020) Lei Sheng et al.   NUCLEIC ACIDS RESEARCH

New Oligodeoxynucleotide Derivatives Containing N-(Sulfonyl)-Phosphoramide Groups

(2020) E. A. Burakova et al.   RUSSIAN JOURNAL OF BIOORGANIC CHEMISTRY

Mesyl phosphoramidate backbone modified antisense oligonucleotides targeting miR-21 with enhanced in vivo therapeutic potency

(2020) Olga A. Patutina et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Neutral and negatively charged phosphate modifications alter thermal stability, kinetics of formation and monovalent ion dependence of DNA G-quadruplexes

(2019) Yongdong Su et al.   Chemistry-An Asian Journal

Mesyl phosphoramidate antisense oligonucleotides as an alternative to phosphorothioates with improved biochemical and biological properties

(2019) S. K. Miroshnichenko et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Nonclinical Exon Skipping Studies with 2′-O-Methyl Phosphorothioate Antisense Oligonucleotides in mdx and mdx-utrn−/− Mice Inspired by Clinical Trial Results

(2019) Maaike van Putten et al.   Nucleic Acid Therapeutics

Oligonucleotide–Palladacycle Conjugates as Splice-Correcting Agents

(2019) Madhuri Hande et al.   MOLECULES

Efficient Conjugation to Phosphorothioate Oligonucleotides by Cu-Catalyzed Huisgen 1,3-Dipolar Cycloaddition

(2019) Malgorzata Honcharenko et al.   BIOCONJUGATE CHEMISTRY

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

(2019) Wen Shen et al.   NATURE BIOTECHNOLOGY

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) Wen Shen et al.   NUCLEIC ACIDS RESEARCH

Therapeutic Oligonucleotides: State of the Art

(2018) C.I. Edvard Smith et al.   Annual Review of Pharmacology and Toxicology

Intrathecal administration of nusinersen in adolescent and adult SMA type 2 and 3 patients

(2018) Claudia D. Wurster et al.   JOURNAL OF NEUROLOGY

Cell-Penetrating Peptide Conjugates of Steric Blocking Oligonucleotides as Therapeutics for Neuromuscular Diseases from a Historical Perspective to Current Prospects of Treatment

(2018) Michael J. Gait et al.   Nucleic Acid Therapeutics

FDA-Approved Oligonucleotide Therapies in 2017

(2017) Cy A. Stein et al.   MOLECULAR THERAPY

The chemical evolution of oligonucleotide therapies of clinical utility

(2017) Anastasia Khvorova et al.   NATURE BIOTECHNOLOGY

Cellular uptake and trafficking of antisense oligonucleotides

(2017) Stanley T Crooke et al.   NATURE BIOTECHNOLOGY

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

(2017) Richard S. Finkel et al.   NEW ENGLAND JOURNAL OF MEDICINE

Chemistry, mechanism and clinical status of antisense oligonucleotides and duplex RNAs

(2017) Xiulong Shen et al.   NUCLEIC ACIDS RESEARCH

New oligodeoxyribonucleotide derivatives bearing internucleotide N-tosyl phosphoramidate groups: Synthesis and complementary binding to DNA and RNA

(2017) D. V. Prokhorova et al.   RUSSIAN JOURNAL OF BIOORGANIC CHEMISTRY

New oligodeoxynucleotide derivatives containing N-(methanesulfonyl)-phosphoramidate (mesyl phosphoramidate) internucleotide group

(2017) B. P. Chelobanov et al.   RUSSIAN JOURNAL OF BIOORGANIC CHEMISTRY

FDA Approval of Nusinersen for Spinal Muscular Atrophy Makes 2016 the Year of Splice Modulating Oligonucleotides

(2017) Annemieke Aartsma-Rus   Nucleic Acid Therapeutics

FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga

(2017) Annemieke Aartsma-Rus et al.   Nucleic Acid Therapeutics

Development of Exon Skipping Therapies for Duchenne Muscular Dystrophy: A Critical Review and a Perspective on the Outstanding Issues

(2017) Annemieke Aartsma-Rus et al.   Nucleic Acid Therapeutics

Molecular Mechanisms of Antisense Oligonucleotides

(2017) Stanley T. Crooke   Nucleic Acid Therapeutics

Rational Design of Short Locked Nucleic Acid-Modified 2′- O -Methyl Antisense Oligonucleotides for Efficient Exon-Skipping In Vitro

(2017) Bao T. Le et al.   Molecular Therapy-Nucleic Acids

Hsp90 protein interacts with phosphorothioate oligonucleotides containing hydrophobic 2′-modifications and enhances antisense activity

(2016) Xue-Hai Liang et al.   NUCLEIC ACIDS RESEARCH

Pharmacokinetics, biodistribution and cell uptake of antisense oligonucleotides

(2015) Richard S. Geary et al.   ADVANCED DRUG DELIVERY REVIEWS

Antisense oligonucleotides in therapy for neurodegenerative disorders

(2015) Melvin M. Evers et al.   ADVANCED DRUG DELIVERY REVIEWS

Application of modified antisense oligonucleotides and siRNAs as antiviral drugs

(2015) Anke Wagner et al.   Future Medicinal Chemistry

Identification and characterization of intracellular proteins that bind oligonucleotides with phosphorothioate linkages

(2015) Xue-hai Liang et al.   NUCLEIC ACIDS RESEARCH

2′-Fluoro-modified phosphorothioate oligonucleotide can cause rapid degradation of P54nrb and PSF

(2015) Wen Shen et al.   NUCLEIC ACIDS RESEARCH

Antisense oligonucleotides in cancer

(2014) Daniela Castanotto et al.   CURRENT OPINION IN ONCOLOGY

Exon-Skipping Antisense Oligonucleotides to Correct Missplicing in Neurogenetic Diseases

(2014) Kavitha Siva et al.   Nucleic Acid Therapeutics

Phosphorothioates, Essential Components of Therapeutic Oligonucleotides

(2014) Fritz Eckstein   Nucleic Acid Therapeutics

Hepatotoxic Potential of Therapeutic Oligonucleotides Can Be Predicted from Their Sequence and Modification Pattern

(2013) Peter H. Hagedorn et al.   Nucleic Acid Therapeutics

Receptors, endocytosis, and trafficking: the biological basis of targeted delivery of antisense and siRNA oligonucleotides

(2012) R. L. Juliano et al.   JOURNAL OF DRUG TARGETING

Molecular and phenotypic reassessment of an infrequently used mouse model for spinal muscular atrophy

(2009) Rocky G. Gogliotti et al.   BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS

Amplification efficiency: linking baseline and bias in the analysis of quantitative PCR data

(2009) J. M. Ruijter et al.   NUCLEIC ACIDS RESEARCH