| Autor: |
A. Malerba, P. Klein, H. Bachtarzi, S. A. Jarmin, G. Cordova, A. Ferry, V. Strings, M. Polay Espinoza, K. Mamchaoui, S. C. Blumen, J. Lacau St Guily, V. Mouly, M. Graham, G. Butler-Browne, D. A. Suhy, C. Trollet, G. Dickson |
| Jazyk: |
angličtina |
| Rok vydání: |
2017 |
| Předmět: |
|
| Zdroj: |
Nature Communications, Vol 8, Iss 1, Pp 1-14 (2017) |
| Druh dokumentu: |
article |
| ISSN: |
2041-1723 |
| DOI: |
10.1038/ncomms14848 |
| Popis: |
Oculopharyngeal muscular dystrophy is caused by trinucleotide repeat expansions in thePABPN1gene. Here the authors use AAV-based gene therapy to knockdown the mutant gene and replace it with a wild-type allele, and show effectiveness in mice and in patient cells. |
| Databáze: |
Directory of Open Access Journals |
| Externí odkaz: |
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