PTC124 targets genetic disorders caused by nonsense mutations
By E. Welch, E. Barton, Jin Zhuo, Y. Tomizawa, W. Friesen, P. Trifillis, S. Paushkin, Meenal Patel, C. Trotta, Seongwoo Hwang and 32 more. Nature, 2007
1,151 citations recorded by the source corpus.
Subfields: Molecular Biology
View Paper: https://www.semanticscholar.org/paper/f789f96553707c894981ff8272cdb8e5d3c4464d
How researchers see this paper
The researchers whose trusted networks rank it highest, strongest advocate first. Every researcher gets a different order.
In Molecular Biology
- Fred H. Gage
- M. Marchetto
- Alysson Renato Muotri
- Gene W. Yeo
- Nahum Sonenberg
- Yangling Mu
- David M. Sabatini
- A. Varki
Citations in the corpus
- Translational Control in Cancer. (2018, background): “Clinical introduction of such a small molecule inhibitor for the treatment of Duchenne muscular dystrophy, known as Translarna (ataluren) (Welch et al. 2007; Finkel et al. 2013) could be useful in the oncology setting, although its level of efficacy remains to be established.”
- A model for neural development and treatment of Rett Syndrome using human induced pluripotent stem cells (2010, background): “New drugs with reduced toxicity and enhanced suppression of premature stop codon mutations might be good therapeutic candidates (Nudelman et al., 2009; Welch et al., 2007).”