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Items: 1 to 20 of 141

1.

The benzamide M344, a novel histone deacetylase inhibitor, significantly increases SMN2 RNA/protein levels in spinal muscular atrophy cells.

Riessland M, Brichta L, Hahnen E, Wirth B.

Hum Genet. 2006 Aug;120(1):101-10. Epub 2006 May 25.

PMID:
16724231
2.
3.

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
4.

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
5.

Restoration of SMN function: delivery of a trans-splicing RNA re-directs SMN2 pre-mRNA splicing.

Coady TH, Shababi M, Tullis GE, Lorson CL.

Mol Ther. 2007 Aug;15(8):1471-8. Epub 2007 Jun 5.

6.
7.

Novel aminoglycosides increase SMN levels in spinal muscular atrophy fibroblasts.

Mattis VB, Rai R, Wang J, Chang CW, Coady T, Lorson CL.

Hum Genet. 2006 Nov;120(4):589-601. Epub 2006 Sep 2.

PMID:
16951947
8.

Spinal muscular atrophy and therapeutic prospects.

Wirth B, Brichta L, Hahnen E.

Prog Mol Subcell Biol. 2006;44:109-32.

PMID:
17076267
9.

Stimulating full-length SMN2 expression by delivering bifunctional RNAs via a viral vector.

Baughan T, Shababi M, Coady TH, Dickson AM, Tullis GE, Lorson CL.

Mol Ther. 2006 Jul;14(1):54-62. Epub 2006 Mar 31.

10.

Hydroxyurea enhances SMN2 gene expression in spinal muscular atrophy cells.

Grzeschik SM, Ganta M, Prior TW, Heavlin WD, Wang CH.

Ann Neurol. 2005 Aug;58(2):194-202.

PMID:
16049920
11.

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
12.

Implication of fetal SMN2 expression in type I SMA pathogenesis: protection or pathological gain of function?

Soler-Botija C, Cuscó I, Caselles L, López E, Baiget M, Tizzano EF.

J Neuropathol Exp Neurol. 2005 Mar;64(3):215-23.

PMID:
15804053
13.

Htra2-beta 1 stimulates an exonic splicing enhancer and can restore full-length SMN expression to survival motor neuron 2 (SMN2).

Hofmann Y, Lorson CL, Stamm S, Androphy EJ, Wirth B.

Proc Natl Acad Sci U S A. 2000 Aug 15;97(17):9618-23.

14.

A non-sequence-specific requirement for SMN protein activity: the role of aminoglycosides in inducing elevated SMN protein levels.

Wolstencroft EC, Mattis V, Bajer AA, Young PJ, Lorson CL.

Hum Mol Genet. 2005 May 1;14(9):1199-210. Epub 2005 Mar 24.

PMID:
15790598
15.

Spinal muscular atrophy: from gene to therapy.

Wirth B, Brichta L, Hahnen E.

Semin Pediatr Neurol. 2006 Jun;13(2):121-31. Review.

PMID:
17027862
16.

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
17.

Induction of full-length survival motor neuron by polyphenol botanical compounds.

Sakla MS, Lorson CL.

Hum Genet. 2008 Jan;122(6):635-43. Epub 2007 Oct 26.

PMID:
17962980
18.

Trichostatin A increases SMN expression and survival in a mouse model of spinal muscular atrophy.

Avila AM, Burnett BG, Taye AA, Gabanella F, Knight MA, Hartenstein P, Cizman Z, Di Prospero NA, Pellizzoni L, Fischbeck KH, Sumner CJ.

J Clin Invest. 2007 Mar;117(3):659-71. Epub 2007 Feb 22.

19.

SRp30c-dependent stimulation of survival motor neuron (SMN) exon 7 inclusion is facilitated by a direct interaction with hTra2 beta 1.

Young PJ, DiDonato CJ, Hu D, Kothary R, Androphy EJ, Lorson CL.

Hum Mol Genet. 2002 Mar 1;11(5):577-87.

PMID:
11875052
20.

Salbutamol increases SMN mRNA and protein levels in spinal muscular atrophy cells.

Angelozzi C, Borgo F, Tiziano FD, Martella A, Neri G, Brahe C.

J Med Genet. 2008 Jan;45(1):29-31. Epub 2007 Oct 11.

PMID:
17932121

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