Cargando…

A Lack of Premature Termination Codon Read-Through Efficacy of PTC124 (Ataluren) in a Diverse Array of Reporter Assays

The drug molecule PTC124 (Ataluren) has been described as a read-through agent, capable of suppressing premature termination codons (PTCs) and restoring functional protein production from genes disrupted by nonsense mutations. Following the discovery of PTC124 there was some controversy regarding it...

Descripción completa

Detalles Bibliográficos
Autores principales:	McElroy, Stuart P., Nomura, Toshifumi, Torrie, Leah S., Warbrick, Emma, Gartner, Ulrike, Wood, Gavin, McLean, W. H. Irwin
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Public Library of Science 2013
Materias:	Research Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3692445/ https://www.ncbi.nlm.nih.gov/pubmed/23824517 http://dx.doi.org/10.1371/journal.pbio.1001593

Ejemplares similares

Toward a Rationale for the PTC124 (Ataluren) Promoted Readthrough of Premature Stop Codons: A Computational Approach and GFP-Reporter Cell-Based Assay
por: Lentini, Laura, et al.
Publicado: (2014)

Aminoglycosides, but not PTC124 (Ataluren), rescue nonsense mutations in the leptin receptor and in luciferase reporter genes
por: Bolze, Florian, et al.
Publicado: (2017)

Ataluren—Promising Therapeutic Premature Termination Codon Readthrough Frontrunner
por: Michorowska, Sylwia
Publicado: (2021)

Ataluren suppresses a premature termination codon in an MPS I-H mouse
por: Wang, Dan, et al.
Publicado: (2022)

Ataluren and aminoglycosides stimulate read-through of nonsense codons by orthogonal mechanisms
por: Ng, Martin Y., et al.
Publicado: (2021)

A Simple and Affordable Method to Create Nonsense Mutation Clones of p53 for Studying the Premature Termination Codon Readthrough Activity of PTC124
por: Chen, Chia-Chi, et al.
Publicado: (2023)

Targeting translational read-through of premature termination mutations in BMPR2 with PTC124 for pulmonary arterial hypertension
por: Long, Lu, et al.
Publicado: (2020)

Generation of Premature Termination Codon (PTC)-Harboring Pseudorabies Virus (PRV) via Genetic Code Expansion Technology
por: Wang, Tong-Yun, et al.
Publicado: (2022)

Effect of Ataluren on dystrophin mutations
por: Berger, Joachim, et al.
Publicado: (2020)

Ataluren Pharmacokinetics in Healthy Japanese and Caucasian Subjects
por: Kong, Ronald, et al.
Publicado: (2019)

Caffeine boosts Ataluren's readthrough activity
por: Lentini, Laura, et al.
Publicado: (2019)

Treatment with Ataluren for Duchene Muscular Dystrophy
por: Morkous, Sameh S.
Publicado: (2020)

Ataluren treatment of patients with nonsense mutation dystrophinopathy
por: Bushby, Katharine, et al.
Publicado: (2014)

Long term treatment with ataluren—the Swedish experience
por: Michael, Eva, et al.
Publicado: (2021)

Burkholderia pseudomallei d‐alanine‐d‐alanine ligase; detailed characterisation and assessment of a potential antibiotic drug target
por: Díaz‐Sáez, Laura, et al.
Publicado: (2019)

Clinical potential of ataluren in the treatment of Duchenne muscular dystrophy
por: Namgoong, John Hyun, et al.
Publicado: (2016)

The effect of PTC124 on choroideremia fibroblasts and iPSC-derived RPE raises considerations for therapy
por: Torriano, Simona, et al.
Publicado: (2018)

3D renal modeling classifies BHD as a ciliopathy, possibly responsive to treatment with PTC124
por: Basten, S, et al.
Publicado: (2012)

Formulation and Stability of Ataluren Eye Drop Oily Solution for Aniridia
por: Djayet, Celia, et al.
Publicado: (2020)

Ataluren for the Treatment of Usher Syndrome 2A Caused by Nonsense Mutations
por: Samanta, Ananya, et al.
Publicado: (2019)

A comparative evaluation of NB30, NB54 and PTC124 in translational read-through efficacy for treatment of an USH1C nonsense mutation
por: Goldmann, Tobias, et al.
Publicado: (2012)

Premature termination codons are recognized in the nucleus in a reading-frame-dependent manner
por: Shi, Min, et al.
Publicado: (2015)

Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients
por: McDonald, Craig M, et al.
Publicado: (2021)

Engineered transfer RNAs for suppression of premature termination codons
por: Lueck, John D., et al.
Publicado: (2019)

Investigation of PTC124-mediated translational readthrough in a retinal organoid model of AIPL1-associated Leber congenital amaurosis
por: Leung, Amy, et al.
Publicado: (2022)

MAD-X PTC Integration
por: Schmidt, Frank, et al.
Publicado: (2005)

CHD4 Predicts Aggressiveness in PTC Patients and Promotes Cancer Stemness and EMT in PTC Cells
por: Pratheeshkumar, Poyil, et al.
Publicado: (2021)

Premature termination codons in modern human genomes
por: Fujikura, Kohei
Publicado: (2016)

In vitro metabolism, reaction phenotyping, enzyme kinetics, CYP inhibition and induction potential of ataluren
por: Kong, Ronald, et al.
Publicado: (2020)

Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study
por: Mercuri, Eugenio, et al.
Publicado: (2020)

The unfolded protein response affects readthrough of premature termination codons
por: Oren, Yifat S, et al.
Publicado: (2014)

PTC124 Rescues Nonsense Mutation of Two Tumor Suppressor Genes NOTCH1 and FAT1 to Repress HNSCC Cell Proliferation
por: Wu, Ming-Han, et al.
Publicado: (2022)

Advances in MAD-X using PTC
por: Skowronski, P K, et al.
Publicado: (2007)

PTC_py-ORBIT - Release & Benchmarking
por: HOLMES, Jeffrey A
Publicado: (2014)

Premature Termination Codon Read-Through in the ABCC6 Gene: Potential Treatment for Pseudoxanthoma Elasticum
por: Zhou, Yong, et al.
Publicado: (2013)

Early treatment with Ataluren of a 2-year-old boy with nonsense mutation Duchenne dystrophy
por: Bitetti, Ilaria, et al.
Publicado: (2021)

Use of PTC124 for nonsense suppression therapy targeting BMP4 nonsense variants in vitro and the bmp4(st72) allele in zebrafish
por: Krall, Max, et al.
Publicado: (2019)

The role of nucleotide composition in premature termination codon recognition
por: Zahdeh, Fouad, et al.
Publicado: (2016)

Molecular Genetics in a Cohort of Patients With Concurrent PTC and Melanoma
por: Joshi, Tripti, et al.
Publicado: (2021)

Ataluren for drug‐resistant epilepsy in nonsense variant‐mediated Dravet syndrome and CDKL5 deficiency disorder
por: Devinsky, Orrin, et al.
Publicado: (2021)

Cannot write session to /tmp/vufind_sessions/sess_i09cjiq0lci4dfn5ubkk7hdl6c