Cargando…

Correction: Pathological mechanism and antisense oligonucleotide-mediated rescue of a non-coding variant suppressing factor 9 RNA biogenesis leading to hemophilia B

Detalles Bibliográficos
Autores principales:	Krooss, Simon, Werwitzke, Sonja, Kopp, Johannes, Rovai, Alice, Varnholt, Dirk, Wachs, Amelie S., Goyenvalle, Aurelie, Aartsma-Rus, Annemieke, Ott, Michael, Tiede, Andreas, Langemeier, Jörg, Bohne, Jens
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Public Library of Science 2021
Materias:	Correction
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7842914/ https://www.ncbi.nlm.nih.gov/pubmed/33507897 http://dx.doi.org/10.1371/journal.pgen.1009345

Ejemplares similares

Pathological mechanism and antisense oligonucleotide-mediated rescue of a non-coding variant suppressing factor 9 RNA biogenesis leading to hemophilia B
por: Krooss, Simon, et al.
Publicado: (2020)

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

Efficient SMN Rescue following Subcutaneous Tricyclo-DNA Antisense Oligonucleotide Treatment
por: Robin, Valérie, et al.
Publicado: (2017)

DMD antisense oligonucleotide mediated exon skipping efficiency correlates with flanking intron retention time and target position within the exon
por: Goossens, Remko, et al.
Publicado: (2023)

Histone deacetylase inhibitors improve antisense-mediated exon-skipping efficacy in mdx mice
por: Bizot, Flavien, et al.
Publicado: (2022)

Therapeutic Potential of Tricyclo-DNA antisense oligonucleotides
por: Goyenvalle, Aurelie, et al.
Publicado: (2016)

Nonclinical Exon Skipping Studies with 2′-O-Methyl Phosphorothioate Antisense Oligonucleotides in mdx and mdx-utrn−/− Mice Inspired by Clinical Trial Results
por: van Putten, Maaike, et al.
Publicado: (2019)

Targeting Several CAG Expansion Diseases by a Single Antisense Oligonucleotide
por: Evers, Melvin M., et al.
Publicado: (2011)

A dystrophic Duchenne mouse model for testing human antisense oligonucleotides
por: Veltrop, Marcel, et al.
Publicado: (2018)

Preparing n-of-1 Antisense Oligonucleotide Treatments for Rare Neurological Diseases in Europe: Genetic, Regulatory, and Ethical Perspectives
por: Synofzik, Matthis, et al.
Publicado: (2022)

Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N-of-1 Exon Skipping Antisense Oligonucleotides
por: Aartsma-Rus, Annemieke, et al.
Publicado: (2023)

Long-term Exon Skipping Studies With 2′-O-Methyl Phosphorothioate Antisense Oligonucleotides in Dystrophic Mouse Models
por: Tanganyika-de Winter, Christa L, et al.
Publicado: (2012)

Considerations in the Preclinical Assessment of the Safety of Antisense Oligonucleotides
por: Goyenvalle, Aurélie, et al.
Publicado: (2023)

Evaluation of 2'-Deoxy-2'-fluoro Antisense Oligonucleotides for Exon Skipping in Duchenne Muscular Dystrophy
por: Jirka, Silvana M G, et al.
Publicado: (2015)

The Effect of 6-Thioguanine on Alternative Splicing and Antisense-Mediated Exon Skipping Treatment for Duchenne Muscular Dystrophy
por: Verhaart, Ingrid E. C., et al.
Publicado: (2012)

Targeting TGF-β Signaling by Antisense Oligonucleotide-mediated Knockdown of TGF-β Type I Receptor
por: Kemaladewi, Dwi U, et al.
Publicado: (2014)

Preclinical Studies on Intestinal Administration of Antisense Oligonucleotides as a Model for Oral Delivery for Treatment of Duchenne Muscular Dystrophy
por: van Putten, Maaike, et al.
Publicado: (2014)

Cyclic Peptides to Improve Delivery and Exon Skipping of Antisense Oligonucleotides in a Mouse Model for Duchenne Muscular Dystrophy
por: Jirka, Silvana M.G., et al.
Publicado: (2018)

Preclinical PK and PD Studies on 2′-O-Methyl-phosphorothioate RNA Antisense Oligonucleotides in the mdx Mouse Model
por: Heemskerk, Hans, et al.
Publicado: (2010)

Next Generation Exon 51 Skipping Antisense Oligonucleotides for Duchenne Muscular Dystrophy
por: van Deutekom, Judith, et al.
Publicado: (2023)

Antisense-Oligonucleotide Mediated Exon Skipping in Activin-Receptor-Like Kinase 2: Inhibiting the Receptor That Is Overactive in Fibrodysplasia Ossificans Progressiva
por: Shi, SongTing, et al.
Publicado: (2013)

The Dynamics of Compound, Transcript, and Protein Effects After Treatment With 2OMePS Antisense Oligonucleotides in mdx Mice
por: Verhaart, Ingrid E C, et al.
Publicado: (2014)

Antisense-induced exon skipping for duplications in Duchenne muscular dystrophy
por: Aartsma-Rus, Annemieke, et al.
Publicado: (2007)

Measuring DNA hybridization using fluorescent DNA-stabilized silver clusters to investigate mismatch effects on therapeutic oligonucleotides
por: de Bruin, Donny, et al.
Publicado: (2018)

von Willebrand factor modulates immune complexes and the recall response against factor VIII in a murine hemophilia A model
por: Oleshko, Olga, et al.
Publicado: (2023)

Inhibition of IL-1 Signaling by Antisense Oligonucleotide-mediated Exon Skipping of IL-1 Receptor Accessory Protein (IL-1RAcP)
por: Yılmaz-Eliş, A Seda, et al.
Publicado: (2013)

Antisense-Mediated RNA Targeting: Versatile and Expedient Genetic Manipulation in the Brain
por: Zalachoras, Ioannis, et al.
Publicado: (2011)

Efficacy and Safety Profile of Tricyclo-DNA Antisense Oligonucleotides in Duchenne Muscular Dystrophy Mouse Model
por: Relizani, Karima, et al.
Publicado: (2017)

Rescue of Outer Hair Cells with Antisense Oligonucleotides in Usher Mice Is Dependent on Age of Treatment
por: Ponnath, Abhilash, et al.
Publicado: (2017)

Novel Ex Vivo Culture Method for the Study of Dupuytren's Disease: Effects of TGFβ Type 1 Receptor Modulation by Antisense Oligonucleotides
por: Karkampouna, Sofia, et al.
Publicado: (2014)

A multicenter comparison of quantification methods for antisense oligonucleotide-induced DMD exon 51 skipping in Duchenne muscular dystrophy cell cultures
por: Hiller, Monika, et al.
Publicado: (2018)

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)

7′,5′-alpha-bicyclo-DNA: new chemistry for oligonucleotide exon splicing modulation therapy
por: Evéquoz, Damien, et al.
Publicado: (2021)

Delivery is key: lessons learnt from developing splice‐switching antisense therapies
por: Godfrey, Caroline, et al.
Publicado: (2017)

Good news for the mdx mouse community: Improved dystrophin restoration after skipping mouse dystrophin exon 23
por: Aartsma-Rus, Annemieke
Publicado: (2022)

Antisense Oligonucleotide-Based Rescue of Aberrant Splicing Defects Caused by 15 Pathogenic Variants in ABCA4
por: Tomkiewicz, Tomasz Z., et al.
Publicado: (2021)

Antisense Oligonucleotide Rescue of Deep-Intronic Variants Activating Pseudoexons in the 6-Pyruvoyl-Tetrahydropterin Synthase Gene
por: Martínez-Pizarro, Ainhoa, et al.
Publicado: (2022)

Cannot write session to /tmp/vufind_sessions/sess_19eldn8cn3m4vc8v0oeesk81nf