Therapeutic exon 'switching' for dysferlinopathies?
作者信息
Lévy Nicolas, Wein Nicolas, Barthelemy Florian, Mouly Vincent, Garcia Luis, Krahn Martin, Bartoli Marc
出版信息
Eur J Hum Genet. 2010 Sep;18(9):969-70; author reply 971. doi: 10.1038/ejhg.2010.73. Epub 2010 May 26.
Abstract
摘要
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本文引用的文献
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Therapeutic exon skipping for dysferlinopathies?
Eur J Hum Genet. 2010 Aug;18(8):889-94. doi: 10.1038/ejhg.2010.4. Epub 2010 Feb 10.
2
Efficient bypass of mutations in dysferlin deficient patient cells by antisense-induced exon skipping.
Hum Mutat. 2010 Feb;31(2):136-42. doi: 10.1002/humu.21160.
3
Progress in therapeutic antisense applications for neuromuscular disorders.
Eur J Hum Genet. 2010 Feb;18(2):146-53. doi: 10.1038/ejhg.2009.160. Epub 2009 Oct 7.
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Lariat branch point mutation in the dysferlin gene with mild limb-girdle muscular dystrophy.
Neurology. 2006 Apr 11;66(7):1114-6. doi: 10.1212/01.wnl.0000204358.89303.81.
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