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Targeting Several CAG Expansion Diseases by a Single Antisense Oligonucleotide
To date there are 9 known diseases caused by an expanded polyglutamine repeat, with the most prevalent being Huntington's disease. Huntington's disease is a progressive autosomal dominant neurodegenerative disorder for which currently no therapy is available. It is caused by a CAG repeat e...
Autores principales: | Evers, Melvin M., Pepers, Barry A., van Deutekom, Judith C. T., Mulders, Susan A. M., den Dunnen, Johan T., Aartsma-Rus, Annemieke, van Ommen, Gert-Jan B., van Roon-Mom, Willeke M. C. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Public Library of Science
2011
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3164722/ https://www.ncbi.nlm.nih.gov/pubmed/21909428 http://dx.doi.org/10.1371/journal.pone.0024308 |
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