4.3 Article

2′-O-(N-(Aminoethyl)carbamoyl)methyl Modification Allows for Lower Phosphorothioate Content in Splice-Switching Oligonucleotides with Retained Activity

相关参考文献

注意:仅列出部分参考文献,下载原文获取全部文献信息。
Article Biochemistry & Molecular Biology

The 10th Oligonucleotide Therapy Approved: Golodirsen for Duchenne Muscular Dystrophy

Annemieke Aartsma-Rus et al.

NUCLEIC ACID THERAPEUTICS (2020)

Article Biochemical Research Methods

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

Malgorzata Honcharenko et al.

BIOCONJUGATE CHEMISTRY (2019)

Article Biotechnology & Applied Microbiology

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

Wen Shen et al.

NATURE BIOTECHNOLOGY (2019)

Review Pharmacology & Pharmacy

Therapeutic Oligonucleotides: State of the Art

C. I. Edvard Smith et al.

ANNUAL REVIEW OF PHARMACOLOGY AND TOXICOLOGY, VOL 59 (2019)

Review Biochemical Research Methods

Bioconjugated Oligonucleotides: Recent Developments and Therapeutic Applications

Sebastien Benizri et al.

BIOCONJUGATE CHEMISTRY (2019)

Article Chemistry, Medicinal

Effective gene silencing activity of prodrug-type 2'-O-methyldithiomethyl siRNA compared with non-prodrug-type 2'-O-methyl siRNA

Junsuke Hayashi et al.

BIOORGANIC & MEDICINAL CHEMISTRY LETTERS (2018)

Review Biotechnology & Applied Microbiology

The chemical evolution of oligonucleotide therapies of clinical utility

Anastasia Khvorova et al.

NATURE BIOTECHNOLOGY (2017)

Editorial Material Medicine, General & Internal

Oligonucleotide Therapeutics - A New Class of Cholesterol-Lowering Drugs

Anastasia Khvorova

NEW ENGLAND JOURNAL OF MEDICINE (2017)

Article Biochemistry & Molecular Biology

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

Annemieke Aartsma-Rus

NUCLEIC ACID THERAPEUTICS (2017)

Article Biochemistry & Molecular Biology

The Effects of 2′-O-Methoxyethyl Containing Antisense Oligonucleotides on Platelets in Human Clinical Trials

Stanley T. Crooke et al.

NUCLEIC ACID THERAPEUTICS (2017)

Review Pharmacology & Pharmacy

Therapeutic nucleic acids: current clinical status

Kannan Sridharan et al.

BRITISH JOURNAL OF CLINICAL PHARMACOLOGY (2016)

Review Peripheral Vascular Disease

Development of Antisense Drugs for Dyslipidemia

Tsuyoshi Yamamoto et al.

JOURNAL OF ATHEROSCLEROSIS AND THROMBOSIS (2016)

Editorial Material Endocrinology & Metabolism

The case for eteplirsen: Paving the way for precision medicine

M. Carrie Miceli et al.

MOLECULAR GENETICS AND METABOLISM (2016)

Editorial Material Biotechnology & Applied Microbiology

New Momentum for the Field of Oligonucleotide Therapeutics

Annemieke Aartsma-Rus

MOLECULAR THERAPY (2016)

Article Biochemistry & Molecular Biology

The delivery of therapeutic oligonucleotides

Rudolph L. Juliano

NUCLEIC ACIDS RESEARCH (2016)

Article Biochemistry & Molecular Biology

Four Novel Splice-Switch Reporter Cell Lines: Distinct Impact of Oligonucleotide Chemistry and Delivery Vector on Biological Activity

Cristina S. J. Rocha et al.

NUCLEIC ACID THERAPEUTICS (2016)

Review Pharmacology & Pharmacy

Cellular uptake and intracellular trafficking of oligonucleotides

R. L. Juliano et al.

ADVANCED DRUG DELIVERY REVIEWS (2015)

Review Pharmacology & Pharmacy

Delivery of oligonucleotides with lipid nanoparticles

Yuhua Wang et al.

ADVANCED DRUG DELIVERY REVIEWS (2015)

Review Pharmacology & Pharmacy

Delivery of therapeutic oligonucleotides with cell penetrating peptides

Prisca Boisguerin et al.

ADVANCED DRUG DELIVERY REVIEWS (2015)

Article Biochemical Research Methods

Optimized luciferase assay for cell-penetrating peptide-mediated delivery of short oligonucleotides

Henrik Helmfors et al.

ANALYTICAL BIOCHEMISTRY (2015)

Article Chemistry, Multidisciplinary

Nuclease resistant oligonucleotides with cell penetrating properties

Stefan Milton et al.

CHEMICAL COMMUNICATIONS (2015)

Review Biotechnology & Applied Microbiology

Oligonucleotide Therapies: The Past and the Present

Karin E. Lundin et al.

HUMAN GENE THERAPY (2015)

Review Genetics & Heredity

Knocking down disease: a progress report on siRNA therapeutics

Anders Wittrup et al.

NATURE REVIEWS GENETICS (2015)

Article Biochemistry & Molecular Biology

Ca2+ enrichment in culture medium potentiates effect of oligonucleotides

Shin-ichiro Hori et al.

NUCLEIC ACIDS RESEARCH (2015)

Review Chemistry, Medicinal

Oligonucleotide delivery: a patent review (2010-2013)

Santiago Grijalvo et al.

EXPERT OPINION ON THERAPEUTIC PATENTS (2014)

Article Biotechnology & Applied Microbiology

Development of Therapeutic Splice-Switching Oligonucleotides

Petra Disterer et al.

HUMAN GENE THERAPY (2014)

Article Medicine, Research & Experimental

Splice-correcting oligonucleotides restore BTK function in X-linked agammaglobulinemia model

Burcu Bestas et al.

JOURNAL OF CLINICAL INVESTIGATION (2014)

Article Biotechnology & Applied Microbiology

Efficient delivery of RNAi prodrugs containing reversible charge-neutralizing phosphotriester backbone modifications

Bryan R. Meade et al.

NATURE BIOTECHNOLOGY (2014)

Review Biochemistry & Molecular Biology

Antisense oligonucleotides: modifications and clinical trials

Vivek K. Sharma et al.

MEDCHEMCOMM (2014)

Review Biochemistry & Molecular Biology

Phosphorothioates, Essential Components of Therapeutic Oligonucleotides

Fritz Eckstein

NUCLEIC ACID THERAPEUTICS (2014)

Article Chemistry, Organic

Synthesis and Stability of a 2′-O-[N-(Aminoethyl)carbamoyl]methyladenosine-Containing Dinucleotide

Stefan Milton et al.

EUROPEAN JOURNAL OF ORGANIC CHEMISTRY (2013)

Article Biochemistry & Molecular Biology

Design, synthesis and biological evaluation of novel antagonist compounds of Toll-like receptors 7, 8 and 9

Ekambar R. Kandimalla et al.

NUCLEIC ACIDS RESEARCH (2013)

Review Chemistry, Medicinal

Nanoparticle-Based Delivery of RNAi Therapeutics: Progress and Challenges

Jiehua Zhou et al.

PHARMACEUTICALS (2013)

Review Biochemistry & Molecular Biology

Designing Chemically Modified Oligonucleotides for Targeted Gene Silencing

Glen F. Deleavey et al.

CHEMISTRY & BIOLOGY (2012)

Review Biotechnology & Applied Microbiology

RNA therapeutics: beyond RNA interference and antisense oligonucleotides

Ryszard Kole et al.

NATURE REVIEWS DRUG DISCOVERY (2012)

Review Biochemistry & Molecular Biology

Special delivery: targeted therapy with small RNAs

D. Peer et al.

GENE THERAPY (2011)

Article Biochemistry & Molecular Biology

An activated triple bond linker enables 'click' attachment of peptides to oligonucleotides on solid support

Malgorzata Wenska et al.

NUCLEIC ACIDS RESEARCH (2011)

Review Genetics & Heredity

Genetic therapies for RNA mis-splicing diseases

Suzan M. Hammond et al.

TRENDS IN GENETICS (2011)

Article Chemistry, Organic

Synthesis of isotopically labeled P-site substrates for the ribosomal peptidyl transferase reaction

Minghong Zhong et al.

JOURNAL OF ORGANIC CHEMISTRY (2008)

Review Chemistry, Medicinal

Chemical modifications to improve the cellular uptake of oligonucleotides

Francoise Debart et al.

CURRENT TOPICS IN MEDICINAL CHEMISTRY (2007)

Article Biochemistry & Molecular Biology

Uptake and quantification of intracellular concentration of lipophilic pro-oligonucleotides in HeLa cells

JC Bologna et al.

ANTISENSE & NUCLEIC ACID DRUG DEVELOPMENT (2002)

Article Biochemistry & Molecular Biology

A novel enzymatic method for the production of purine-2′-deoxyribonucleosides

K Yokozeki et al.

JOURNAL OF MOLECULAR CATALYSIS B-ENZYMATIC (2000)