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Items: 5

1.

A large animal model of spinal muscular atrophy and correction of phenotype.

Duque SI, Arnold WD, Odermatt P, Li X, Porensky PN, Schmelzer L, Meyer K, Kolb SJ, Schümperli D, Kaspar BK, Burghes AH.

Ann Neurol. 2015 Mar;77(3):399-414. doi: 10.1002/ana.24332. Epub 2015 Feb 9.

2.

Electrophysiological Biomarkers in Spinal Muscular Atrophy: Preclinical Proof of Concept.

Arnold WD, Porensky PN, McGovern VL, Iyer CC, Duque S, Li X, Meyer K, Schmelzer L, Kaspar BK, Kolb SJ, Kissel JT, Burghes AH.

Ann Clin Transl Neurol. 2014 Jan 1;1(1):34-44.

3.

Improved antisense oligonucleotide design to suppress aberrant SMN2 gene transcript processing: towards a treatment for spinal muscular atrophy.

Mitrpant C, Porensky P, Zhou H, Price L, Muntoni F, Fletcher S, Wilton SD, Burghes AH.

PLoS One. 2013 Apr 22;8(4):e62114. doi: 10.1371/journal.pone.0062114. Print 2013.

4.

Antisense oligonucleotides for the treatment of spinal muscular atrophy.

Porensky PN, Burghes AH.

Hum Gene Ther. 2013 May;24(5):489-98. doi: 10.1089/hum.2012.225. Review.

5.

A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse.

Porensky PN, Mitrpant C, McGovern VL, Bevan AK, Foust KD, Kaspar BK, Wilton SD, Burghes AH.

Hum Mol Genet. 2012 Apr 1;21(7):1625-38. doi: 10.1093/hmg/ddr600. Epub 2011 Dec 20.

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