Targeting DMPK with Antisense Oligonucleotide Improves Muscle Strength in Myotonic Dystrophy Type 1 Mice

Myotonic dystrophy type 1 (DM1), a dominant hereditary muscular dystrophy, is caused by an abnormal expansion of a (CTG)(n) trinucleotide repeat in the 3′ UTR of the human dystrophia myotonica protein kinase (DMPK) gene. As a consequence, mutant transcripts containing expanded CUG repeats are retain...

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Publicat a:Mol Ther Nucleic Acids
Autors principals: Jauvin, Dominic, Chrétien, Jessina, Pandey, Sanjay K., Martineau, Laurie, Revillod, Lucille, Bassez, Guillaume, Lachon, Aline, McLeod, A. Robert, Gourdon, Geneviève, Wheeler, Thurman M., Thornton, Charles A., Bennett, C. Frank, Puymirat, Jack
Format: Artigo
Idioma:Inglês
Publicat: American Society of Gene & Cell Therapy 2017
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Original Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC5453865/
https://ncbi.nlm.nih.gov/pubmed/28624222
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2017.05.007
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