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...

Descripción completa

Detalles Bibliográficos
Autores principales: Thada, Vaughn, Miller, Jake N., Kovács, Attila D., Pearce, David A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2015
Materias:
Short Communication
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

Internet

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

Ejemplares similares