Considerations in the Preclinical Assessment of the Safety of Antisense Oligonucleotides-Reference-Cited by-同舟云学术

Considerations in the Preclinical Assessment of the Safety of Antisense Oligonucleotides

Published:2023-02-01 Issue:1 Volume:33 Page:1-16
ISSN:2159-3337
Container-title:Nucleic Acid Therapeutics
language:en
Short-container-title:Nucleic Acid Therapeutics

Author:

Goyenvalle Aurélie¹^ORCID,Jimenez-Mallebrera Cecilia²³⁴,van Roon Willeke⁵^ORCID,Sewing Sabine⁶,Krieg Arthur M.⁷,Arechavala-Gomeza Virginia⁸⁹^ORCID,Andersson Patrik¹⁰^ORCID

Affiliation:

1. Université Paris-Saclay, UVSQ, Inserm, END-ICAP, Versailles, France.

2. Laboratorio de Investigación Aplicada en Enfermedades Neuromusculares, Unidad de Patología Neuromuscular, Servicio de Neuropediatría, Institut de Recerca Sant Joan de Déu, Esplugues de Llobregat, Spain.

3. Centro de Investigaciones Biomédicas en Red de Enfermedades Raras (CIBERER), Madrid, Spain.

4. Departamento de Genética, Microbiología y Estadística, Universitat de Barcelona, Barcelona, Spain.

5. Department of Human Genetics, Leiden University Medical Center, Leiden, the Netherlands.

6. Pharma Research and Early Development, Roche Innovation Center Basel, Basel, Switzerland.

7. RNA Therapeutics Institute, University of Massachusetts, Worcester, Massachusetts, USA.

8. Neuromuscular Disorders, Biocruces Bizkaia Health Research Institute, Barakaldo, Spain.

9. Ikerbasque, Basque Foundation for Science, Bilbao, Spain.

10. Safety Innovation, Safety Sciences, Clinical Pharmacology and Safety Sciences, R&D, AstraZeneca, Gothenburg, Sweden.

Publisher

Mary Ann Liebert Inc

Subject

Drug Discovery,Genetics,Molecular Biology,Molecular Medicine,Biochemistry

Link

https://www.liebertpub.com/doi/pdf/10.1089/nat.2022.0061

Reference146 articles.

1. Principles for targeting RNA with drug-like small molecules

2. Antisense RNA Therapeutics: A Brief Overview

3. Nonsense-mediated mRNA decay: splicing, translation and mRNP dynamics

4. Antisense oligonucleotide–directed inhibition of nonsense-mediated mRNA decay

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