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32329 A novel mouse model of Ataxia Telangiectasia for testing small molecule readthrough compounds
ABSTRACT IMPACT: Small molecule readthrough compounds are a promising therapeutic with the potential to overcome nonsense mutations thereby enabling the production of functional ATM protein in patients with Ataxia Telangiectasia OBJECTIVES/GOALS: To generate a novel mouse model of Ataxia-Telangiecta...
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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Cambridge University Press
2021
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8827764/ http://dx.doi.org/10.1017/cts.2021.430 |