ArticleNature communications2013
SMRT compounds abrogate cellular phenotypes of ataxia telangiectasia in neural derivatives of patient-specific hiPSCs.
Article in Nature communications, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
21 citing papers in PubMed, 40 citations in OpenAlex.
- Therapeutic Potential of Translational Readthrough at Disease-Associated Premature Termination Codons From Tumor Suppressor Genes.IUBMB life · 2025Review
- Amlexanox: Readthrough Induction and Nonsense-Mediated mRNA Decay Inhibition in a Charcot-Marie-Tooth Model of hiPSCs-Derived Neuronal Cells Harboring a Nonsense Mutation inPharmaceuticals (Basel, Switzerland) · 2023Article
- Cerebellar glutamatergic system impacts spontaneous motor recovery by regulating Gria1 expression.NPJ Regenerative medicine · 2022Article
- Bone Marrow Transplantation as Therapy for Ataxia-Telangiectasia: A Systematic Review.Cancers · 2020Review
- CIRM tools and technologies: Breaking bottlenecks to the development of stem cell therapies.Stem cells translational medicine · 2020Article
- Chromosome instability syndromes.Nature reviews. Disease primers · 2019Review
- ATM deficiency promotes progression of CRPC by enhancing Warburg effect.Endocrine-related cancer · 2019Article
- Reconstitution of the Ataxia-Telangiectasia Cellular Phenotype With Lentiviral Vectors.Frontiers in immunology · 2018Article
- Nijmegen Breakage Syndrome fibroblasts and iPSCs: cellular models for uncovering disease-associated signaling pathways and establishing a screening platform for anti-oxidants.Scientific reports · 2017Article
- Induced pluripotent stem cells from patients with focal cortical dysplasia and refractory epilepsy.Molecular medicine reports · 2017Article
- Influence of ATM-Mediated DNA Damage Response on Genomic Variation in Human Induced Pluripotent Stem Cells.Stem cells and development · 2016Article
- Pluripotent stem cells in disease modelling and drug discovery.Nature reviews. Molecular cell biology · 2016Review
- Utility of Induced Pluripotent Stem Cells for the Study and Treatment of Genetic Diseases: Focus on Childhood Neurological Disorders.Frontiers in molecular neuroscience · 2016Review
- Spontaneous ATM Gene Reversion in A-T iPSC to Produce an Isogenic Cell Line.Stem cell reports · 2015Article
- Induced Pluripotent Stem Cell Models to Enable In Vitro Models for Screening in the Central Nervous System.Stem cells and development · 2015Review
- Probing disorders of the nervous system using reprogramming approaches.The EMBO journal · 2015Review
- Application of biomaterials to advance induced pluripotent stem cell research and therapy.The EMBO journal · 2015Review
- Stem cells: the pursuit of genomic stability.International journal of molecular sciences · 2014Review
- Ataxia telangiectasia derived iPS cells show preserved x-ray sensitivity and decreased chromosomal instability.Scientific reports · 2014Article
- A new series of small molecular weight compounds induce read through of all three types of nonsense mutations in the ATM gene.Molecular therapy : the journal of the American Society of Gene Therapy · 2013Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
10 authors at 3 institutions in 2 countries.
Funding
Abstract
Ataxia telangiectasia is a devastating neurodegenerative disease caused primarily by loss of function mutations in ATM, a hierarchical DNA repair gene and tumour suppressor. So far, murine models of ataxia telangiectasia have failed to accurately recapitulate many aspects of the disease, most notably, the progressive cerebellar ataxia. Here we present a model of human ataxia telangiectasia using induced pluripotent stem cells, and show that small molecule read-through compounds, designed to induce read-through of mRNA around premature termination codons, restore ATM activity and improve the response to DNA damage. This platform allows for efficient screening of novel compounds, identification of target and off-target effects, and preclinical testing on relevant cell types for the pathogenic dissection and treatment of ataxia telangiectasia.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.