Cargando…

Improvements in the Tolerability Profile of 2′-O-Methoxyethyl Chimeric Antisense Oligonucleotides in Parallel with Advances in Design, Screening, and Other Methods

The development process of antisense oligonucleotides (ASOs) as therapeutic agents in humans has advanced through the implementation of chemical compound modifications as well as increasingly sophisticated toxicological preclinical screening techniques. The Ionis Integrated Safety Database was utili...

Descripción completa

Detalles Bibliográficos
Autores principales:	Partridge, Wesley, Xia, Shuting, Kwoh, T. Jesse, Bhanot, Sanjay, Geary, Richard S., Baker, Brenda F.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Mary Ann Liebert, Inc., publishers 2021
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8713270/ https://www.ncbi.nlm.nih.gov/pubmed/34242101 http://dx.doi.org/10.1089/nat.2020.0917

Ejemplares similares

Integrated Assessment of Phase 2 Data on GalNAc(3)-Conjugated 2′-O-Methoxyethyl-Modified Antisense Oligonucleotides
por: Baker, Brenda F., et al.
Publicado: (2023)

Integrated Safety Assessment of 2′-O-Methoxyethyl Chimeric Antisense Oligonucleotides in NonHuman Primates and Healthy Human Volunteers
por: Crooke, Stanley T, et al.
Publicado: (2016)

The Effects of 2′-O-Methoxyethyl Containing Antisense Oligonucleotides on Platelets in Human Clinical Trials
por: Crooke, Stanley T., et al.
Publicado: (2017)

The Effects of 2′-O-Methoxyethyl Oligonucleotides on Renal Function in Humans
por: Crooke, Stanley T., et al.
Publicado: (2018)

Integrated Assessment of the Clinical Performance of GalNAc(3)-Conjugated 2′-O-Methoxyethyl Chimeric Antisense Oligonucleotides: I. Human Volunteer Experience
por: Crooke, Stanley T., et al.
Publicado: (2019)

Correlations between preclinical BJAB assay ranking of antisense drugs and clinical trial adverse events
por: Partridge, Wesley, et al.
Publicado: (2023)

Effective Exon Skipping and Dystrophin Restoration by 2′-O-Methoxyethyl Antisense Oligonucleotide in Dystrophin-Deficient Mice
por: Yang, Lu, et al.
Publicado: (2013)

Predictive Dose-Based Estimation of Systemic Exposure Multiples in Mouse and Monkey Relative to Human for Antisense Oligonucleotides With 2′-O-(2-Methoxyethyl) Modifications
por: Yu, Rosie Z, et al.
Publicado: (2015)

Treatment with Volanesorsen, a 2′-O-Methoxyethyl-Modified Antisense Oligonucleotide Targeting APOC3 mRNA, Does Not Affect the QTc Interval in Healthy Volunteers
por: Watts, Lynnetta M., et al.
Publicado: (2020)

Chimeric Antisense Oligonucleotide Conjugated to α-Tocopherol
por: Nishina, Tomoko, et al.
Publicado: (2015)

Lack of QT Prolongation for 2′-O-Methoxyethyl-Modified Antisense Oligonucleotides Based on Retrospective Exposure/Response Analysis of Ten Phase 1 Dose-Escalation Placebo-Controlled Studies in Healthy Subjects
por: Yu, Rosie Z., et al.
Publicado: (2017)

Clinical and Preclinical Pharmacokinetics and Pharmacodynamics of Mipomersen (Kynamro(®)): A Second-Generation Antisense Oligonucleotide Inhibitor of Apolipoprotein B
por: Geary, Richard S., et al.
Publicado: (2015)

Efficient Synthesis of 2’‐O‐Methoxyethyl Oligonucleotide‐Cationic Peptide Conjugates
por: Halloy, François, et al.
Publicado: (2021)

Solid-Phase Synthesis of Boranophosphate/Phosphorothioate/Phosphate Chimeric Oligonucleotides and Their Potential as Antisense Oligonucleotides
por: Takahashi, Yuhei, et al.
Publicado: (2021)

Sequence-specific 2'-O-methoxyethyl antisense oligonucleotides activate human platelets through glycoprotein VI, triggering formation of platelet-leukocyte aggregates
por: Slingsby, Martina H. Lundberg, et al.
Publicado: (2021)

Systematic evaluation of 2′-Fluoro modified chimeric antisense oligonucleotide-mediated exon skipping in vitro
por: Chen, Suxiang, et al.
Publicado: (2019)

Safety, tolerability and antiviral activity of the antisense oligonucleotide bepirovirsen in patients with chronic hepatitis B: a phase 2 randomized controlled trial
por: Yuen, Man-Fung, et al.
Publicado: (2021)

Peptide Conjugates of a 2′-O-Methoxyethyl Phosphorothioate Splice-Switching Oligonucleotide Show Increased Entrapment in Endosomes
por: Hill, Alyssa C., et al.
Publicado: (2023)

Ligand conjugated antisense oligonucleotide for the treatment of transthyretin amyloidosis: preclinical and phase 1 data
por: Viney, Nicholas J., et al.
Publicado: (2020)

Enhancement of SMN2 Exon 7 Inclusion by Antisense Oligonucleotides Targeting the Exon
por: Hua, Yimin, et al.
Publicado: (2007)

Antisense oligonucleotides in neurological disorders
por: Wurster, Claudia D., et al.
Publicado: (2018)

Molecular Mechanisms of Antisense Oligonucleotides
por: Crooke, Stanley T.
Publicado: (2017)

Antisense oligonucleotides: A primer
por: Scoles, Daniel R., et al.
Publicado: (2019)

Antisense Oligonucleotides and RNA Interference
por: Kher, Gitanjali, et al.
Publicado: (2011)

N-(2-Methoxyethyl)phthalimide
por: Sim, Yoke Leng, et al.
Publicado: (2008)

PK/PD modeling of FXI antisense oligonucleotides to bridge the dose‐FXI activity relation from healthy volunteers to end‐stage renal disease patients
por: Willmann, Stefan, et al.
Publicado: (2021)

Transthyretin Antisense Oligonucleotides Lower Circulating RBP4 Levels and Improve Insulin Sensitivity in Obese Mice
por: Zemany, Laura, et al.
Publicado: (2015)

Author Correction: Systematic evaluation of 2′-Fluoro modified chimeric antisense oligonucleotide-mediated exon skipping in vitro
por: Chen, Suxiang, et al.
Publicado: (2020)

Peripheral Reduction of FGFR4 with Antisense Oligonucleotides Increases Metabolic Rate and Lowers Adiposity in Diet-Induced Obese Mice
por: Yu, Xing Xian, et al.
Publicado: (2013)

Opportunities and challenges for antisense oligonucleotide therapies
por: Kuijper, Elsa C., et al.
Publicado: (2020)

Silencing Antibiotic Resistance with Antisense Oligonucleotides
por: Jani, Saumya, et al.
Publicado: (2021)

Drug Discovery Perspectives of Antisense Oligonucleotides
por: Kim, Yeonjoon
Publicado: (2023)

In-silico study of antisense oligonucleotide antibiotics
por: Chen, Erica S., et al.
Publicado: (2023)

A randomised, open-label, parallel group phase 2 study of antisense oligonucleotide therapy in acromegaly
por: Trainer, Peter J, et al.
Publicado: (2018)

Targeting FMN, TPP, SAM-I, and glmS Riboswitches with Chimeric Antisense Oligonucleotides for Completely Rational Antibacterial Drug Development
por: Pavlova, Nikolet, et al.
Publicado: (2023)

Preclinical and Phase 1 Assessment of Antisense Oligonucleotide Bepirovirsen in Hepatitis B Virus–Transgenic Mice and Healthy Human Volunteers: Support for Clinical Dose Selection and Evaluation of Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses
por: Han, Kelong, et al.
Publicado: (2022)

Reduction of Off-Target Effects of Gapmer Antisense Oligonucleotides by Oligonucleotide Extension
por: Yasuhara, Hidenori, et al.
Publicado: (2022)

Targeting density-enhanced phosphatase-1 (DEP-1) with antisense oligonucleotides improves the metabolic phenotype in high-fat diet-fed mice
por: Krüger, Janine, et al.
Publicado: (2013)

Towards next generation antisense oligonucleotides: mesylphosphoramidate modification improves therapeutic index and duration of effect of gapmer antisense oligonucleotides
por: Anderson, Brooke A, et al.
Publicado: (2021)

Population Pharmacokinetic–Pharmacodynamic Modeling of Inotersen, an Antisense Oligonucleotide for Treatment of Patients with Hereditary Transthyretin Amyloidosis
por: Yu, Rosie Z., et al.
Publicado: (2020)

Cannot write session to /tmp/vufind_sessions/sess_495sln2lor8edvngjecbn2v6ac