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Tissue‐specific variation in nonsense mutant transcript level and drug‐induced read‐through efficiency in the Cln1 (R151X) mouse model of INCL
About 10% of inherited diseases are caused by nonsense mutations [Trends Mol Med 18 (2012) 688], and nonsense suppression drug therapy promoting translation through premature stop codons is an emerging therapeutic approach. Infantile neuronal ceroid lipofuscinosis (INCL), a childhood neurodegenerati...
| Autores principales: | , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
John Wiley and Sons Inc.
2015
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4727554/ https://www.ncbi.nlm.nih.gov/pubmed/26648046 http://dx.doi.org/10.1111/jcmm.12744 |