The DOI matches its Crossref record; 1 of the 1 references it cites were found in a registry or an index.
PTC124 targets genetic disorders caused by nonsense mutations
Overview
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Integrity signals
Registry
Cites
Statistics
Verification
Claims the paper makes
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Self-check
Percentages, sums and reported differences re-computed from the paper’s own numbers.
The self-check needs the paper’s full text.
Citations
Cites
Nonsense-mediated mRNA decay: splicing, translation and mRNP dynamics
Maquat, L. E. (2004) · Nature Reviews Molecular Cell Biology · doi:10.1038/nrm1310 · Cited by 1,214
Cited by
Revisiting the role of ABC transporters in multidrug-resistant cancer
Robey, R. W., Pluchino, K. M., Hall, M. D., Fojo, A. T., Bates, S. E., & Gottesman, M. M. (2018) · Nature Reviews Cancer · doi:10.1038/s41568-018-0005-8 · Cited by 1,866
Marchetto, M. C. N., Carromeu, C., Acab, A., Yu, D., Yeo, G. W., Mu, Y., Chen, G., Gage, F. H., & Muotri, A. R. (2010) · Cell · doi:10.1016/j.cell.2010.10.016 · Cited by 1,329
Self-citationO'Sullivan, B. P., & Freedman, S. D. (2009) · The Lancet · doi:10.1016/s0140-6736(09)60327-5 · Cited by 1,320
A functional CFTR assay using primary cystic fibrosis intestinal organoids
Dekkers, J. F., Wiegerinck, C. L., de Jonge, H. R., Bronsveld, I., Janssens, H. M., de Winter-de Groot, K. M., Brandsma, A. M., de Jong, N. W. M., Bijvelds, M. J. C., Scholte, B. J., et al. (2013) · Nature Medicine · doi:10.1038/nm.3201 · Cited by 999
Targeting mutant p53 for efficient cancer therapy
Bykov, V. J. N., Eriksson, S. E., Bianchi, J., & Wiman, K. G. (2018) · Nature Reviews Cancer · doi:10.1038/nrc.2017.109 · Cited by 889
Maguire, A. M., High, K. A., Auricchio, A., Wright, J. F., Pierce, E. A., Testa, F., Mingozzi, F., Bennicelli, J. L., Ying, G.-S., Rossi, S., et al. (2009) · The Lancet · doi:10.1016/s0140-6736(09)61836-5 · Cited by 865
NMD: a multifaceted response to premature translational termination
Kervestin, S., & Jacobson, A. (2012) · Nature Reviews Molecular Cell Biology · doi:10.1038/nrm3454 · Cited by 572
Self-citationThe evolutionary consequences of erroneous protein synthesis
Allan Drummond, D., & Wilke, C. O. (2009) · Nature Reviews Genetics · doi:10.1038/nrg2662 · Cited by 515
The discovery of first-in-class drugs: origins and evolution
Eder, J., Sedrani, R., & Wiesmann, C. (2014) · Nature Reviews Drug Discovery · doi:10.1038/nrd4336 · Cited by 514
Nonsense-mediated mRNA decay in humans at a glance
Kurosaki, T., & Maquat, L. E. (2016) · Journal of Cell Science · doi:10.1242/jcs.181008 · Cited by 396
Therapies for rare diseases: therapeutic modalities, progress and challenges ahead
Tambuyzer, E., Vandendriessche, B., Austin, C. P., Brooks, P. J., Larsson, K., Miller Needleman, K. I., Valentine, J., Davies, K., Groft, S. C., Preti, R., et al. (2020) · Nature Reviews Drug Discovery · doi:10.1038/s41573-019-0049-9 · Cited by 357
RNA Quality Control in Eukaryotes
Doma, M. K., & Parker, R. (2007) · Cell · doi:10.1016/j.cell.2007.10.041 · Cited by 345
Mutations in SYNGAP1 in Autosomal Nonsyndromic Mental Retardation
Hamdan, F. F., Gauthier, J., Spiegelman, D., Noreau, A., Yang, Y., Pellerin, S., Dobrzeniecka, S., Côté, M., Perreau-Linck, E., Carmant, L., et al. (2009) · New England Journal of Medicine · doi:10.1056/nejmoa0805392 · Cited by 336
Therapeutics Based on Stop Codon Readthrough
Keeling, K. M., Xue, X., Gunn, G., & Bedwell, D. M. (2014) · Annual Review of Genomics and Human Genetics · doi:10.1146/annurev-genom-091212-153527 · Cited by 306
The ribosomal basis of diamond-blackfan anemia: mutation and database update
Boria, I., Garelli, E., Gazda, H. T., Aspesi, A., Quarello, P., Pavesi, E., Ferrante, D., Meerpohl, J. J., Kartal, M., Da Costa, L., et al. (2010) · Human Mutation · doi:10.1002/humu.21383 · Cited by 233
Pluripotent stem cells in neurodegenerative and neurodevelopmental diseases
Marchetto, M. C. N., Winner, B., & Gage, F. H. (2010) · Human Molecular Genetics · doi:10.1093/hmg/ddq159 · Cited by 152
Neu-Yilik, G., Amthor, B., Gehring, N. H., Bahri, S., Paidassi, H., Hentze, M. W., & Kulozik, A. E. (2011) · RNA · doi:10.1261/rna.2401811 · Cited by 140
Shimizu-Motohashi, Y., Komaki, H., Motohashi, N., Takeda, S., Yokota, T., & Aoki, Y. (2019) · Journal of Personalized Medicine · doi:10.3390/jpm9010001 · Cited by 82
Molecular Therapies for Inherited Retinal Diseases—Current Standing, Opportunities and Challenges
Vázquez-Domínguez, I., Garanto, A., & Collin, R. W. J. (2019) · Genes · doi:10.3390/genes10090654 · Cited by 80
Skeletal Muscle Degenerative Diseases and Strategies for Therapeutic Muscle Repair
Tabebordbar, M., Wang, E. T., & Wagers, A. J. (2013) · Annual Review of Pathology: Mechanisms of Disease · doi:10.1146/annurev-pathol-011811-132450 · Cited by 79
Reversibility of functional deficits in experimental models of Rett syndrome
Cobb, S., Guy, J., & Bird, A. (2010) · Biochemical Society Transactions · doi:10.1042/bst0380498 · Cited by 67
Succinic Semialdehyde Dehydrogenase Deficiency: An Update
Didiasova, M., Banning, A., Brennenstuhl, H., Jung-Klawitter, S., Cinquemani, C., Opladen, T., & Tikkanen, R. (2020) · Cells · doi:10.3390/cells9020477 · Cited by 52
MicroRNA-33 inhibition ameliorates muscular dystrophy by enhancing skeletal muscle regeneration
Sowa, N., Horie, T., Ide, Y., Baba, O., Kora, K., Yoshida, T., Nakamura, Y., Matsumura, S., Matsushita, K., Imanaka, M., et al. (2025) · EMBO Molecular Medicine · doi:10.1038/s44321-025-00273-9 · Cited by 4
Metadata
- Authors
- Welch, E. M., Barton, E. R., Zhuo, J., Tomizawa, Y., Friesen, W. J., Trifillis, P., Paushkin, S., Patel, M., Trotta, C. R., Hwang, S., Wilde, R. G., Karp, G., Takasugi, J., Chen, G., Jones, S., Ren, H., Moon, Y.-C., Corson, D., Turpoff, A. A., Campbell, J. A., Conn, M. M., Khan, A., Almstead, N. G., Hedrick, J., Mollin, A., Risher, N., Weetall, M., Yeh, S., Branstrom, A. A., Colacino, J. M., Babiak, J., Ju, W. D., Hirawat, S., Northcutt, V. J., Miller, L. L., Spatrick, P., He, F., Kawana, M., Feng, H., Jacobson, A., Peltz, S. W., & Sweeney, H. L.
- Venue
- Nature
- Published
- 2007-04-22
- Volume
- 447
- Issue
- 7140
- Pages
- 87-91
- ISSN
- 0028-0836 (ISSN-L)
- 1476-4687
- OpenAlex
- W2085811948
- Cited by
- 1,137
- References
- 1
- FWCI
- 30.41
- Type
- journal article
- Language
- en
- Open access
- closed
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
Are you an author, or do you see a problem with this page? Request a review
Data sources
Bibliographic data: Semantic Scholar, OpenAlex, Crossref.
Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
Semantic Scholar’s data is described in: Kinney, R., et al. (2023). The Semantic Scholar Open Data Platform. arXiv:2301.10140.
