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Items: 5

1.

Antisense Oligonucleotides Used to Target the DUX4 mRNA as Therapeutic Approaches in FaciosScapuloHumeral Muscular Dystrophy (FSHD).

Ansseau E, Vanderplanck C, Wauters A, Harper SQ, Coppée F, Belayew A.

Genes (Basel). 2017 Mar 3;8(3). pii: E93. doi: 10.3390/genes8030093.

2.

TSUNAMI: an antisense method to phenocopy splicing-associated diseases in animals.

Sahashi K, Hua Y, Ling KK, Hung G, Rigo F, Horev G, Katsuno M, Sobue G, Ko CP, Bennett CF, Krainer AR.

Genes Dev. 2012 Aug 15;26(16):1874-84. doi: 10.1101/gad.197418.112.

3.

Multiple exon skipping strategies to by-pass dystrophin mutations.

Adkin CF, Meloni PL, Fletcher S, Adams AM, Muntoni F, Wong B, Wilton SD.

Neuromuscul Disord. 2012 Apr;22(4):297-305. doi: 10.1016/j.nmd.2011.10.007. Epub 2011 Dec 17.

4.

The FSHD atrophic myotube phenotype is caused by DUX4 expression.

Vanderplanck C, Ansseau E, Charron S, Stricwant N, Tassin A, Laoudj-Chenivesse D, Wilton SD, Coppée F, Belayew A.

PLoS One. 2011;6(10):e26820. doi: 10.1371/journal.pone.0026820. Epub 2011 Oct 28.

5.

Current status of pharmaceutical and genetic therapeutic approaches to treat DMD.

Pichavant C, Aartsma-Rus A, Clemens PR, Davies KE, Dickson G, Takeda S, Wilton SD, Wolff JA, Wooddell CI, Xiao X, Tremblay JP.

Mol Ther. 2011 May;19(5):830-40. doi: 10.1038/mt.2011.59. Epub 2011 Apr 5. Review.

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