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Items: 1 to 20 of 154

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.

Repeated low doses of morpholino antisense oligomer: an intermediate mouse model of spinal muscular atrophy to explore the window of therapeutic response.

Zhou H, Meng J, Marrosu E, Janghra N, Morgan J, Muntoni F.

Hum Mol Genet. 2015 Nov 15;24(22):6265-77. doi: 10.1093/hmg/ddv329. Epub 2015 Aug 11.

8.
9.

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.

10.

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.

11.

Spinal muscular atrophy phenotype is ameliorated in human motor neurons by SMN increase via different novel RNA therapeutic approaches.

Nizzardo M, Simone C, Dametti S, Salani S, Ulzi G, Pagliarani S, Rizzo F, Frattini E, Pagani F, Bresolin N, Comi G, Corti S.

Sci Rep. 2015 Jun 30;5:11746. doi: 10.1038/srep11746.

12.

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.

13.

Investigation of New Morpholino Oligomers to Increase Survival Motor Neuron Protein Levels in Spinal Muscular Atrophy.

Ramirez A, Crisafulli SG, Rizzuti M, Bresolin N, Comi GP, Corti S, Nizzardo M.

Int J Mol Sci. 2018 Jan 6;19(1). pii: E167. doi: 10.3390/ijms19010167.

14.

Normalization of Patient-Identified Plasma Biomarkers in SMNΔ7 Mice following Postnatal SMN Restoration.

Arnold WD, Duque S, Iyer CC, Zaworski P, McGovern VL, Taylor SJ, von Herrmann KM, Kobayashi DT, Chen KS, Kolb SJ, Paushkin SV, Burghes AH.

PLoS One. 2016 Dec 1;11(12):e0167077. doi: 10.1371/journal.pone.0167077. eCollection 2016.

15.

Mechanistic principles of antisense targets for the treatment of spinal muscular atrophy.

Singh NN, Lee BM, DiDonato CJ, Singh RN.

Future Med Chem. 2015;7(13):1793-808. doi: 10.4155/fmc.15.101. Epub 2015 Sep 18. Review.

16.

Optimization of Morpholino Antisense Oligonucleotides Targeting the Intronic Repressor Element1 in Spinal Muscular Atrophy.

Osman EY, Washington CW 3rd, Kaifer KA, Mazzasette C, Patitucci TN, Florea KM, Simon ME, Ko CP, Ebert AD, Lorson CL.

Mol Ther. 2016 Sep;24(9):1592-601. doi: 10.1038/mt.2016.145. Epub 2016 Jul 9.

17.

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.

18.

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.

19.

Combination of valproic acid and morpholino splice-switching oligonucleotide produces improved outcomes in spinal muscular atrophy patient-derived fibroblasts.

Farrelly-Rosch A, Lau CL, Patil N, Turner BJ, Shabanpoor F.

Neurochem Int. 2017 Sep;108:213-221. doi: 10.1016/j.neuint.2017.02.016. Epub 2017 Apr 4.

PMID:
28389270
20.

Histopathological Defects in Intestine in Severe Spinal Muscular Atrophy Mice Are Improved by Systemic Antisense Oligonucleotide Treatment.

Sintusek P, Catapano F, Angkathunkayul N, Marrosu E, Parson SH, Morgan JE, Muntoni F, Zhou H.

PLoS One. 2016 May 10;11(5):e0155032. doi: 10.1371/journal.pone.0155032. eCollection 2016.

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