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Items: 1 to 20 of 126

1.

Effect of combined systemic and local morpholino treatment on the spinal muscular atrophy Δ7 mouse model phenotype.

Nizzardo M, Simone C, Salani S, Ruepp MD, Rizzo F, Ruggieri M, Zanetta C, Brajkovic S, Moulton HM, Müehlemann O, Bresolin N, Comi GP, Corti S.

Clin Ther. 2014 Mar 1;36(3):340-56.e5. doi: 10.1016/j.clinthera.2014.02.004.

PMID:
24636820
2.

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.

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.

4.

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.

5.

Morpholino antisense oligonucleotides targeting intronic repressor Element1 improve phenotype in SMA mouse models.

Osman EY, Miller MR, Robbins KL, Lombardi AM, Atkinson AK, Brehm AJ, Lorson CL.

Hum Mol Genet. 2014 Sep 15;23(18):4832-45. doi: 10.1093/hmg/ddu198. Epub 2014 Apr 29.

6.

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.

7.
8.

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.

9.

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.

10.

Rescue of gene-expression changes in an induced mouse model of spinal muscular atrophy by an antisense oligonucleotide that promotes inclusion of SMN2 exon 7.

Staropoli JF, Li H, Chun SJ, Allaire N, Cullen P, Thai A, Fleet CM, Hua Y, Bennett CF, Krainer AR, Kerr D, McCampbell A, Rigo F, Carulli JP.

Genomics. 2015 Apr;105(4):220-8. doi: 10.1016/j.ygeno.2015.01.007. Epub 2015 Jan 31.

11.

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.

12.

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.

13.

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
14.

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.

15.
16.

Splicing regulation in spinal muscular atrophy by an RNA structure formed by long-distance interactions.

Singh NN, Lee BM, Singh RN.

Ann N Y Acad Sci. 2015 Apr;1341:176-87. doi: 10.1111/nyas.12727. Epub 2015 Feb 27.

17.

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.

18.

Antisense masking of an hnRNP A1/A2 intronic splicing silencer corrects SMN2 splicing in transgenic mice.

Hua Y, Vickers TA, Okunola HL, Bennett CF, Krainer AR.

Am J Hum Genet. 2008 Apr;82(4):834-48. doi: 10.1016/j.ajhg.2008.01.014. Epub 2008 Mar 27.

19.

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.

20.

SMN deficiency alters Nrxn2 expression and splicing in zebrafish and mouse models of spinal muscular atrophy.

See K, Yadav P, Giegerich M, Cheong PS, Graf M, Vyas H, Lee SG, Mathavan S, Fischer U, Sendtner M, Winkler C.

Hum Mol Genet. 2014 Apr 1;23(7):1754-70. doi: 10.1093/hmg/ddt567. Epub 2013 Nov 11.

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