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Items: 1 to 20 of 50

1.

Early heart failure in the SMNDelta7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN delivery.

Bevan AK, Hutchinson KR, Foust KD, Braun L, McGovern VL, Schmelzer L, Ward JG, Petruska JC, Lucchesi PA, Burghes AH, Kaspar BK.

Hum Mol Genet. 2010 Oct 15;19(20):3895-905. doi: 10.1093/hmg/ddq300. Epub 2010 Jul 16.

2.

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.

3.

Systemic delivery of scAAV9 expressing SMN prolongs survival in a model of spinal muscular atrophy.

Valori CF, Ning K, Wyles M, Mead RJ, Grierson AJ, Shaw PJ, Azzouz M.

Sci Transl Med. 2010 Jun 9;2(35):35ra42. doi: 10.1126/scitranslmed.3000830.

4.

Spinal muscular atrophy: mechanisms and therapeutic strategies.

Lorson CL, Rindt H, Shababi M.

Hum Mol Genet. 2010 Apr 15;19(R1):R111-8. doi: 10.1093/hmg/ddq147. Epub 2010 Apr 13. Review.

5.

CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy.

Passini MA, Bu J, Roskelley EM, Richards AM, Sardi SP, O'Riordan CR, Klinger KW, Shihabuddin LS, Cheng SH.

J Clin Invest. 2010 Apr;120(4):1253-64. doi: 10.1172/JCI41615. Epub 2010 Mar 15.

6.

Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN.

Foust KD, Wang X, McGovern VL, Braun L, Bevan AK, Haidet AM, Le TT, Morales PR, Rich MM, Burghes AH, Kaspar BK.

Nat Biotechnol. 2010 Mar;28(3):271-4. doi: 10.1038/nbt.1610. Epub 2010 Feb 28.

7.

Tetracyclines that promote SMN2 exon 7 splicing as therapeutics for spinal muscular atrophy.

Hastings ML, Berniac J, Liu YH, Abato P, Jodelka FM, Barthel L, Kumar S, Dudley C, Nelson M, Larson K, Edmonds J, Bowser T, Draper M, Higgins P, Krainer AR.

Sci Transl Med. 2009 Nov 4;1(5):5ra12. doi: 10.1126/scitranslmed.3000208.

8.

SAHA ameliorates the SMA phenotype in two mouse models for spinal muscular atrophy.

Riessland M, Ackermann B, Förster A, Jakubik M, Hauke J, Garbes L, Fritzsche I, Mende Y, Blumcke I, Hahnen E, Wirth B.

Hum Mol Genet. 2010 Apr 15;19(8):1492-506. doi: 10.1093/hmg/ddq023. Epub 2010 Jan 22.

PMID:
20097677
9.

RNA targeting therapeutics: molecular mechanisms of antisense oligonucleotides as a therapeutic platform.

Bennett CF, Swayze EE.

Annu Rev Pharmacol Toxicol. 2010;50:259-93. doi: 10.1146/annurev.pharmtox.010909.105654. Review.

PMID:
20055705
10.

Trans-splicing-mediated improvement in a severe mouse model of spinal muscular atrophy.

Coady TH, Lorson CL.

J Neurosci. 2010 Jan 6;30(1):126-30. doi: 10.1523/JNEUROSCI.4489-09.2010.

11.

Embryonic stem cell-derived neural stem cells improve spinal muscular atrophy phenotype in mice.

Corti S, Nizzardo M, Nardini M, Donadoni C, Salani S, Ronchi D, Simone C, Falcone M, Papadimitriou D, Locatelli F, Mezzina N, Gianni F, Bresolin N, Comi GP.

Brain. 2010 Feb;133(Pt 2):465-81. doi: 10.1093/brain/awp318. Epub 2009 Dec 23.

PMID:
20032086
12.

Effects of 2,4-diaminoquinazoline derivatives on SMN expression and phenotype in a mouse model for spinal muscular atrophy.

Butchbach ME, Singh J, Thorsteinsdóttir M, Saieva L, Slominski E, Thurmond J, Andrésson T, Zhang J, Edwards JD, Simard LR, Pellizzoni L, Jarecki J, Burghes AH, Gurney ME.

Hum Mol Genet. 2010 Feb 1;19(3):454-67. doi: 10.1093/hmg/ddp510. Epub 2009 Nov 6.

13.

Delivery of a read-through inducing compound, TC007, lessens the severity of a spinal muscular atrophy animal model.

Mattis VB, Ebert AD, Fosso MY, Chang CW, Lorson CL.

Hum Mol Genet. 2009 Oct 15;18(20):3906-13. doi: 10.1093/hmg/ddp333. Epub 2009 Jul 21.

14.

LBH589 induces up to 10-fold SMN protein levels by several independent mechanisms and is effective even in cells from SMA patients non-responsive to valproate.

Garbes L, Riessland M, Hölker I, Heller R, Hauke J, Tränkle C, Coras R, Blümcke I, Hahnen E, Wirth B.

Hum Mol Genet. 2009 Oct 1;18(19):3645-58. doi: 10.1093/hmg/ddp313. Epub 2009 Jul 7.

PMID:
19584083
15.

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.

16.

Phase II open label study of valproic acid in spinal muscular atrophy.

Swoboda KJ, Scott CB, Reyna SP, Prior TW, LaSalle B, Sorenson SL, Wood J, Acsadi G, Crawford TO, Kissel JT, Krosschell KJ, D'Anjou G, Bromberg MB, Schroth MK, Chan GM, Elsheikh B, Simard LR.

PLoS One. 2009;4(5):e5268. doi: 10.1371/journal.pone.0005268. Epub 2009 May 14.

17.

A short antisense oligonucleotide masking a unique intronic motif prevents skipping of a critical exon in spinal muscular atrophy.

Singh NN, Shishimorova M, Cao LC, Gangwani L, Singh RN.

RNA Biol. 2009 Jul-Aug;6(3):341-50. Epub 2009 Jul 14.

18.

Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons.

Duque S, Joussemet B, Riviere C, Marais T, Dubreil L, Douar AM, Fyfe J, Moullier P, Colle MA, Barkats M.

Mol Ther. 2009 Jul;17(7):1187-96. doi: 10.1038/mt.2009.71. Epub 2009 Apr 14.

19.

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.

20.

Spinal muscular atrophy.

Oskoui M, Kaufmann P.

Neurotherapeutics. 2008 Oct;5(4):499-506. doi: 10.1016/j.nurt.2008.08.007. Review.

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