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Items: 1 to 20 of 77

1.

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.

2.

In vitro and in vivo effects of 2,4 diaminoquinazoline inhibitors of the decapping scavenger enzyme DcpS: Context-specific modulation of SMN transcript levels.

Cherry JJ, DiDonato CJ, Androphy EJ, Calo A, Potter K, Custer SK, Du S, Foley TL, Gopalsamy A, Reedich EJ, Gordo SM, Gordon W, Hosea N, Jones LH, Krizay DK, LaRosa G, Li H, Mathur S, Menard CA, Patel P, Ramos-Zayas R, Rietz A, Rong H, Zhang B, Tones MA.

PLoS One. 2017 Sep 25;12(9):e0185079. doi: 10.1371/journal.pone.0185079. eCollection 2017.

3.

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.

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.

Design of Potent mRNA Decapping Scavenger Enzyme (DcpS) Inhibitors with Improved Physicochemical Properties To Investigate the Mechanism of Therapeutic Benefit in Spinal Muscular Atrophy (SMA).

Gopalsamy A, Narayanan A, Liu S, Parikh MD, Kyne RE Jr, Fadeyi O, Tones MA, Cherry JJ, Nabhan JF, LaRosa G, Petersen DN, Menard C, Foley TL, Noell S, Ren Y, Loria PM, Maglich-Goodwin J, Rong H, Jones LH.

J Med Chem. 2017 Apr 13;60(7):3094-3108. doi: 10.1021/acs.jmedchem.7b00124. Epub 2017 Mar 16.

PMID:
28257199
6.

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.

7.

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.

8.

The effect of the DcpS inhibitor D156844 on the protective action of follistatin in mice with spinal muscular atrophy.

Harris AW, Butchbach ME.

Neuromuscul Disord. 2015 Sep;25(9):699-705. doi: 10.1016/j.nmd.2015.05.008. Epub 2015 May 27.

9.

Decapping Scavenger (DcpS) enzyme: advances in its structure, activity and roles in the cap-dependent mRNA metabolism.

Milac AL, Bojarska E, Wypijewska del Nogal A.

Biochim Biophys Acta. 2014 Jun;1839(6):452-62. doi: 10.1016/j.bbagrm.2014.04.007. Epub 2014 Apr 15. Review.

PMID:
24742626
10.

DcpS is a transcript-specific modulator of RNA in mammalian cells.

Zhou M, Bail S, Plasterer HL, Rusche J, Kiledjian M.

RNA. 2015 Jul;21(7):1306-12. doi: 10.1261/rna.051573.115. Epub 2015 May 22.

11.

Diverse small-molecule modulators of SMN expression found by high-throughput compound screening: early leads towards a therapeutic for spinal muscular atrophy.

Jarecki J, Chen X, Bernardino A, Coovert DD, Whitney M, Burghes A, Stack J, Pollok BA.

Hum Mol Genet. 2005 Jul 15;14(14):2003-18. Epub 2005 Jun 8.

PMID:
15944201
12.

Combination of SMN2 copy number and NAIP deletion predicts disease severity in spinal muscular atrophy.

Watihayati MS, Fatemeh H, Marini M, Atif AB, Zahiruddin WM, Sasongko TH, Tang TH, Zabidi-Hussin ZA, Nishio H, Zilfalil BA.

Brain Dev. 2009 Jan;31(1):42-5. doi: 10.1016/j.braindev.2008.08.012. Epub 2008 Oct 7.

PMID:
18842367
13.

Molecular and functional analysis of intragenic SMN1 mutations in patients with spinal muscular atrophy.

Sun Y, Grimmler M, Schwarzer V, Schoenen F, Fischer U, Wirth B.

Hum Mutat. 2005 Jan;25(1):64-71.

PMID:
15580564
14.

A library approach to rapidly discover photoaffinity probes of the mRNA decapping scavenger enzyme DcpS.

Xu H, Hett EC, Gopalsamy A, Parikh MD, Geoghegan KF, Kyne RE Jr, Menard CA, Narayanan A, Robinson RP, Johnson DS, Tones MA, Jones LH.

Mol Biosyst. 2015 Oct;11(10):2709-12. doi: 10.1039/c5mb00288e.

PMID:
25959423
15.

The effect of diet on the protective action of D156844 observed in spinal muscular atrophy mice.

Butchbach ME, Singh J, Gurney ME, Burghes AH.

Exp Neurol. 2014 Jun;256:1-6. doi: 10.1016/j.expneurol.2014.03.005. Epub 2014 Mar 25.

16.

Valproic acid increases the SMN2 protein level: a well-known drug as a potential therapy for spinal muscular atrophy.

Brichta L, Hofmann Y, Hahnen E, Siebzehnrubl FA, Raschke H, Blumcke I, Eyupoglu IY, Wirth B.

Hum Mol Genet. 2003 Oct 1;12(19):2481-9. Epub 2003 Jul 29.

PMID:
12915451
17.

[Mutation analysis of SMN1 gene in patients with spinal muscular atrophy].

DU J, Qu YJ, Xiong H, Li EZ, Jin YW, Bai JL, Wang H, Song F.

Zhonghua Er Ke Za Zhi. 2011 Jun;49(6):411-5. Chinese.

PMID:
21924051
18.

Correlation between SMN2 copy number and clinical phenotype of spinal muscular atrophy: three SMN2 copies fail to rescue some patients from the disease severity.

Harada Y, Sutomo R, Sadewa AH, Akutsu T, Takeshima Y, Wada H, Matsuo M, Nishio H.

J Neurol. 2002 Sep;249(9):1211-9.

PMID:
12242541
19.

Therapeutics development for spinal muscular atrophy.

Sumner CJ.

NeuroRx. 2006 Apr;3(2):235-45. Review.

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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