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Results: 1 to 20 of 121

1.

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.

PMID:
22186025
[PubMed - indexed for MEDLINE]
Free PMC Article
2.

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.

PMID:
23630626
[PubMed - indexed for MEDLINE]
Free PMC Article
3.

Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model.

Hua Y, Sahashi K, Rigo F, Hung G, Horev G, Bennett CF, Krainer AR.

Nature. 2011 Oct 5;478(7367):123-6. doi: 10.1038/nature10485.

PMID:
21979052
[PubMed - indexed for MEDLINE]
Free PMC Article
4.

Oligonucleotide-mediated survival of motor neuron protein expression in CNS improves phenotype in a mouse model of spinal muscular atrophy.

Williams JH, Schray RC, Patterson CA, Ayitey SO, Tallent MK, Lutz GJ.

J Neurosci. 2009 Jun 17;29(24):7633-8. doi: 10.1523/JNEUROSCI.0950-09.2009.

PMID:
19535574
[PubMed - indexed for MEDLINE]
Free Article
5.

A novel morpholino oligomer targeting ISS-N1 improves rescue of severe spinal muscular atrophy transgenic mice.

Zhou H, Janghra N, Mitrpant C, Dickinson RL, Anthony K, Price L, Eperon IC, Wilton SD, Morgan J, Muntoni F.

Hum Gene Ther. 2013 Mar;24(3):331-42. doi: 10.1089/hum.2012.211. Epub 2013 Mar 6.

PMID:
23339722
[PubMed - indexed for MEDLINE]
Free PMC Article
6.

Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model.

Hua Y, Sahashi K, Hung G, Rigo F, Passini MA, Bennett CF, Krainer AR.

Genes Dev. 2010 Aug 1;24(15):1634-44. doi: 10.1101/gad.1941310. Epub 2010 Jul 12.

PMID:
20624852
[PubMed - indexed for MEDLINE]
Free PMC Article
7.

SMNDelta7, the major product of the centromeric survival motor neuron (SMN2) gene, extends survival in mice with spinal muscular atrophy and associates with full-length SMN.

Le TT, Pham LT, Butchbach ME, Zhang HL, Monani UR, Coovert DD, Gavrilina TO, Xing L, Bassell GJ, Burghes AH.

Hum Mol Genet. 2005 Mar 15;14(6):845-57. Epub 2005 Feb 9.

PMID:
15703193
[PubMed - indexed for MEDLINE]
Free Article
8.

Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy.

Passini MA, Bu J, Richards AM, Kinnecom C, Sardi SP, Stanek LM, Hua Y, Rigo F, Matson J, Hung G, Kaye EM, Shihabuddin LS, Krainer AR, Bennett CF, Cheng SH.

Sci Transl Med. 2011 Mar 2;3(72):72ra18. doi: 10.1126/scitranslmed.3001777.

PMID:
21368223
[PubMed - indexed for MEDLINE]
Free PMC Article
9.

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.

PMID:
23544870
[PubMed - indexed for MEDLINE]
Free PMC Article
10.

Combination of SMN trans-splicing and a neurotrophic factor increases the life span and body mass in a severe model of spinal muscular atrophy.

Shababi M, Glascock J, Lorson CL.

Hum Gene Ther. 2011 Feb;22(2):135-44. doi: 10.1089/hum.2010.114. Epub 2010 Dec 19.

PMID:
20804424
[PubMed - indexed for MEDLINE]
11.

A negatively acting bifunctional RNA increases survival motor neuron both in vitro and in vivo.

Dickson A, Osman E, Lorson CL.

Hum Gene Ther. 2008 Nov;19(11):1307-15. doi: 10.1089/hgt.2008.067.

PMID:
19848583
[PubMed - indexed for MEDLINE]
Free PMC Article
12.

Antisense oligonucleotides and spinal muscular atrophy: skipping along.

Burghes AH, McGovern VL.

Genes Dev. 2010 Aug 1;24(15):1574-9. doi: 10.1101/gad.1961710.

PMID:
20679391
[PubMed - indexed for MEDLINE]
Free PMC Article
13.

Splicing of a critical exon of human Survival Motor Neuron is regulated by a unique silencer element located in the last intron.

Singh NK, Singh NN, Androphy EJ, Singh RN.

Mol Cell Biol. 2006 Feb;26(4):1333-46.

PMID:
16449646
[PubMed - indexed for MEDLINE]
Free PMC Article
14.

Pathological impact of SMN2 mis-splicing in adult SMA mice.

Sahashi K, Ling KK, Hua Y, Wilkinson JE, Nomakuchi T, Rigo F, Hung G, Xu D, Jiang YP, Lin RZ, Ko CP, Bennett CF, Krainer AR.

EMBO Mol Med. 2013 Oct;5(10):1586-601. doi: 10.1002/emmm.201302567. Epub 2013 Sep 9.

PMID:
24014320
[PubMed - indexed for MEDLINE]
Free PMC Article
15.

5-(N-ethyl-N-isopropyl)-amiloride enhances SMN2 exon 7 inclusion and protein expression in spinal muscular atrophy cells.

Yuo CY, Lin HH, Chang YS, Yang WK, Chang JG.

Ann Neurol. 2008 Jan;63(1):26-34.

PMID:
17924536
[PubMed - indexed for MEDLINE]
16.

Development and characterization of an SMN2-based intermediate mouse model of Spinal Muscular Atrophy.

Cobb MS, Rose FF, Rindt H, Glascock JJ, Shababi M, Miller MR, Osman EY, Yen PF, Garcia ML, Martin BR, Wetz MJ, Mazzasette C, Feng Z, Ko CP, Lorson CL.

Hum Mol Genet. 2013 May 1;22(9):1843-55. doi: 10.1093/hmg/ddt037. Epub 2013 Feb 5.

PMID:
23390132
[PubMed - indexed for MEDLINE]
Free Article
17.

Delivery of bifunctional RNAs that target an intronic repressor and increase SMN levels in an animal model of spinal muscular atrophy.

Baughan TD, Dickson A, Osman EY, Lorson CL.

Hum Mol Genet. 2009 May 1;18(9):1600-11. doi: 10.1093/hmg/ddp076. Epub 2009 Feb 19.

PMID:
19228773
[PubMed - indexed for MEDLINE]
Free PMC Article
18.

Evolving concepts on human SMN pre-mRNA splicing.

Singh RN.

RNA Biol. 2007 Jan-Mar;4(1):7-10. Epub 2007 Jun 4.

PMID:
17592254
[PubMed - indexed for MEDLINE]
Free Article
19.

Decreasing disease severity in symptomatic, Smn(-/-);SMN2(+/+), spinal muscular atrophy mice following scAAV9-SMN delivery.

Glascock JJ, Osman EY, Wetz MJ, Krogman MM, Shababi M, Lorson CL.

Hum Gene Ther. 2012 Mar;23(3):330-5. doi: 10.1089/hum.2011.166. Epub 2012 Jan 26.

PMID:
22029744
[PubMed - indexed for MEDLINE]
Free PMC Article
20.

Bifunctional RNAs targeting the intronic splicing silencer N1 increase SMN levels and reduce disease severity in an animal model of spinal muscular atrophy.

Osman EY, Yen PF, Lorson CL.

Mol Ther. 2012 Jan;20(1):119-26. doi: 10.1038/mt.2011.232. Epub 2011 Oct 25.

PMID:
22031236
[PubMed - indexed for MEDLINE]
Free PMC Article

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