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Items: 1 to 20 of 112

1.

The DcpS inhibitor RG3039 improves survival, function and motor unit pathologies in two SMA mouse models.

Gogliotti RG, Cardona H, Singh J, Bail S, Emery C, Kuntz N, Jorgensen M, Durens M, Xia B, Barlow C, Heier CR, Plasterer HL, Jacques V, Kiledjian M, Jarecki J, Rusche J, DiDonato CJ.

Hum Mol Genet. 2013 Oct 15;22(20):4084-101. doi: 10.1093/hmg/ddt258. Epub 2013 Jun 4.

2.

The DcpS inhibitor RG3039 improves motor function in SMA mice.

Van Meerbeke JP, Gibbs RM, Plasterer HL, Miao W, Feng Z, Lin MY, Rucki AA, Wee CD, Xia B, Sharma S, Jacques V, Li DK, Pellizzoni L, Rusche JR, Ko CP, Sumner CJ.

Hum Mol Genet. 2013 Oct 15;22(20):4074-83. doi: 10.1093/hmg/ddt257. Epub 2013 May 31.

3.

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.

4.

Synthesis and biological evaluation of novel 2,4-diaminoquinazoline derivatives as SMN2 promoter activators for the potential treatment of spinal muscular atrophy.

Thurmond J, Butchbach ME, Palomo M, Pease B, Rao M, Bedell L, Keyvan M, Pai G, Mishra R, Haraldsson M, Andresson T, Bragason G, Thosteinsdottir M, Bjornsson JM, Coovert DD, Burghes AH, Gurney ME, Singh J.

J Med Chem. 2008 Feb 14;51(3):449-69. doi: 10.1021/jm061475p. Epub 2008 Jan 19.

PMID:
18205293
5.

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.

6.

Triptolide increases transcript and protein levels of survival motor neurons in human SMA fibroblasts and improves survival in SMA-like mice.

Hsu YY, Jong YJ, Tsai HH, Tseng YT, An LM, Lo YC.

Br J Pharmacol. 2012 Jun;166(3):1114-26. doi: 10.1111/j.1476-5381.2012.01829.x.

7.

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.

8.

DcpS as a therapeutic target for spinal muscular atrophy.

Singh J, Salcius M, Liu SW, Staker BL, Mishra R, Thurmond J, Michaud G, Mattoon DR, Printen J, Christensen J, Bjornsson JM, Pollok BA, Kiledjian M, Stewart L, Jarecki J, Gurney ME.

ACS Chem Biol. 2008 Nov 21;3(11):711-22. doi: 10.1021/cb800120t.

9.

A SMN missense mutation complements SMN2 restoring snRNPs and rescuing SMA mice.

Workman E, Saieva L, Carrel TL, Crawford TO, Liu D, Lutz C, Beattie CE, Pellizzoni L, Burghes AH.

Hum Mol Genet. 2009 Jun 15;18(12):2215-29. doi: 10.1093/hmg/ddp157. Epub 2009 Mar 27.

10.

The human centromeric survival motor neuron gene (SMN2) rescues embryonic lethality in Smn(-/-) mice and results in a mouse with spinal muscular atrophy.

Monani UR, Sendtner M, Coovert DD, Parsons DW, Andreassi C, Le TT, Jablonka S, Schrank B, Rossoll W, Prior TW, Morris GE, Burghes AH.

Hum Mol Genet. 2000 Feb 12;9(3):333-9. Erratum in: Hum Mol Genet. 2007 Nov 1;16(21):2648. Rossol, W [corrected to Rossoll, W].

11.

Fasudil improves survival and promotes skeletal muscle development in a mouse model of spinal muscular atrophy.

Bowerman M, Murray LM, Boyer JG, Anderson CL, Kothary R.

BMC Med. 2012 Mar 7;10:24. doi: 10.1186/1741-7015-10-24.

12.

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
13.

Motor neuron disease. SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy.

Naryshkin NA, Weetall M, Dakka A, Narasimhan J, Zhao X, Feng Z, Ling KK, Karp GM, Qi H, Woll MG, Chen G, Zhang N, Gabbeta V, Vazirani P, Bhattacharyya A, Furia B, Risher N, Sheedy J, Kong R, Ma J, Turpoff A, Lee CS, Zhang X, Moon YC, Trifillis P, Welch EM, Colacino JM, Babiak J, Almstead NG, Peltz SW, Eng LA, Chen KS, Mull JL, Lynes MS, Rubin LL, Fontoura P, Santarelli L, Haehnke D, McCarthy KD, Schmucki R, Ebeling M, Sivaramakrishnan M, Ko CP, Paushkin SV, Ratni H, Gerlach I, Ghosh A, Metzger F.

Science. 2014 Aug 8;345(6197):688-93. doi: 10.1126/science.1250127.

14.

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.

15.

Hypoxia is a modifier of SMN2 splicing and disease severity in a severe SMA mouse model.

Bebee TW, Dominguez CE, Samadzadeh-Tarighat S, Akehurst KL, Chandler DS.

Hum Mol Genet. 2012 Oct 1;21(19):4301-13. doi: 10.1093/hmg/dds263. Epub 2012 Jul 3.

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.

17.

Enhancement of SMN protein levels in a mouse model of spinal muscular atrophy using novel drug-like compounds.

Cherry JJ, Osman EY, Evans MC, Choi S, Xing X, Cuny GD, Glicksman MA, Lorson CL, Androphy EJ.

EMBO Mol Med. 2013 Jul;5(7):1035-50. doi: 10.1002/emmm.201202305. Epub 2013 Jun 5.

18.

Detection of human survival motor neuron (SMN) protein in mice containing the SMN2 transgene: applicability to preclinical therapy development for spinal muscular atrophy.

Mattis VB, Butchbach ME, Lorson CL.

J Neurosci Methods. 2008 Oct 30;175(1):36-43. doi: 10.1016/j.jneumeth.2008.07.024. Epub 2008 Aug 15.

19.

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.

20.

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.

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