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A novel DMD IRES results in a functional N-truncated dystrophin, providing a potential route to therapy for patients with 5’ mutations

Most mutations that truncate the reading frame of the DMD gene cause loss of dystrophin expression and lead to Duchenne muscular dystrophy. However, amelioration of disease severity can result from alternate translation initiation beginning in DMD exon 6 that leads to expression of a highly function...

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Detalles Bibliográficos
Autores principales:	Wein, Nicolas, Vulin, Adeline, Sofia Falzarano, Maria, Al-Khalili Szigyarto, Christina, Maiti, Baijayanta, Findlay, Andrew, Heller, Kristin N, Uhlén, Mathias, Bakthavachalu, Baskar, Messina, Sonia, Vita, Giuseppe, Passarelli, Chiara, Gualandi, Francesca, Wilton, Steve D, Rodino-Klapac, Louise, Yang, Lin, Dunn, Diane M., Schoenberg, Daniel, Weiss, Robert B., Howard, Michael T., Ferlini, Alessandra, Flanigan, Kevin M.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	2014
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4165597/ https://www.ncbi.nlm.nih.gov/pubmed/25108525 http://dx.doi.org/10.1038/nm.3628

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