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Results: 1 to 20 of 40

1.

Systemic administration of PRO051 in Duchenne's muscular dystrophy.

Goemans NM, Tulinius M, van den Akker JT, Burm BE, Ekhart PF, Heuvelmans N, Holling T, Janson AA, Platenburg GJ, Sipkens JA, Sitsen JM, Aartsma-Rus A, van Ommen GJ, Buyse G, Darin N, Verschuuren JJ, Campion GV, de Kimpe SJ, van Deutekom JC.

N Engl J Med. 2011 Apr 21;364(16):1513-22. doi: 10.1056/NEJMoa1011367. Epub 2011 Mar 23. Erratum in: N Engl J Med. 2011 Oct 6;365(14):1361.

PMID:
21428760
[PubMed - indexed for MEDLINE]
Free Article
2.

Nuclear and cytoplasmic localization of neural stem cell microRNAs.

Jeffries CD, Fried HM, Perkins DO.

RNA. 2011 Apr;17(4):675-86. doi: 10.1261/rna.2006511. Epub 2011 Mar 1.

PMID:
21363885
[PubMed - indexed for MEDLINE]
Free PMC Article
3.

Activation of LDL receptor expression by small RNAs complementary to a noncoding transcript that overlaps the LDLR promoter.

Matsui M, Sakurai F, Elbashir S, Foster DJ, Manoharan M, Corey DR.

Chem Biol. 2010 Dec 22;17(12):1344-55. doi: 10.1016/j.chembiol.2010.10.009.

PMID:
21168770
[PubMed - indexed for MEDLINE]
Free PMC Article
4.

Allele-selective inhibition of huntingtin expression by switching to an miRNA-like RNAi mechanism.

Hu J, Liu J, Corey DR.

Chem Biol. 2010 Nov 24;17(11):1183-8. doi: 10.1016/j.chembiol.2010.10.013.

PMID:
21095568
[PubMed - indexed for MEDLINE]
Free PMC Article
5.

Phosphorylation of human Argonaute proteins affects small RNA binding.

Rüdel S, Wang Y, Lenobel R, Körner R, Hsiao HH, Urlaub H, Patel D, Meister G.

Nucleic Acids Res. 2011 Mar;39(6):2330-43. doi: 10.1093/nar/gkq1032. Epub 2010 Nov 10.

PMID:
21071408
[PubMed - indexed for MEDLINE]
Free PMC Article
6.

The status of exon skipping as a therapeutic approach to duchenne muscular dystrophy.

Lu QL, Yokota T, Takeda S, Garcia L, Muntoni F, Partridge T.

Mol Ther. 2011 Jan;19(1):9-15. doi: 10.1038/mt.2010.219. Epub 2010 Oct 26. Review.

PMID:
20978473
[PubMed - indexed for MEDLINE]
Free PMC Article
7.

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.

PMID:
20679391
[PubMed - indexed for MEDLINE]
Free PMC Article
8.

Involvement of argonaute proteins in gene silencing and activation by RNAs complementary to a non-coding transcript at the progesterone receptor promoter.

Chu Y, Yue X, Younger ST, Janowski BA, Corey DR.

Nucleic Acids Res. 2010 Nov;38(21):7736-48. doi: 10.1093/nar/gkq648. Epub 2010 Jul 30.

PMID:
20675357
[PubMed - indexed for MEDLINE]
Free PMC Article
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.

PMID:
20624852
[PubMed - indexed for MEDLINE]
Free PMC Article
10.

Transcriptional regulation by small RNAs at sequences downstream from 3' gene termini.

Yue X, Schwartz JC, Chu Y, Younger ST, Gagnon KT, Elbashir S, Janowski BA, Corey DR.

Nat Chem Biol. 2010 Aug;6(8):621-9. doi: 10.1038/nchembio.400. Epub 2010 Jun 27.

PMID:
20581822
[PubMed - indexed for MEDLINE]
Free PMC Article
11.

Deep sequencing of human nuclear and cytoplasmic small RNAs reveals an unexpectedly complex subcellular distribution of miRNAs and tRNA 3' trailers.

Liao JY, Ma LM, Guo YH, Zhang YC, Zhou H, Shao P, Chen YQ, Qu LH.

PLoS One. 2010 May 14;5(5):e10563. doi: 10.1371/journal.pone.0010563.

PMID:
20498841
[PubMed - indexed for MEDLINE]
Free PMC Article
12.

Clinical status of duplex RNA.

Watts JK, Corey DR.

Bioorg Med Chem Lett. 2010 Jun 1;20(11):3203-7. doi: 10.1016/j.bmcl.2010.03.109. Epub 2010 Apr 2.

PMID:
20399650
[PubMed - indexed for MEDLINE]
Free PMC Article
13.

Spinal muscular atrophy: mechanisms and therapeutic strategies.

Lorson CL, Rindt H, Shababi M.

Hum Mol Genet. 2010 Apr 15;19(R1):R111-8. doi: 10.1093/hmg/ddq147. Epub 2010 Apr 13. Review.

PMID:
20392710
[PubMed - indexed for MEDLINE]
Free PMC Article
14.

Alternative splicing and evolution: diversification, exon definition and function.

Keren H, Lev-Maor G, Ast G.

Nat Rev Genet. 2010 May;11(5):345-55. doi: 10.1038/nrg2776. Epub 2010 Apr 8. Review.

PMID:
20376054
[PubMed - indexed for MEDLINE]
15.

RNA-targeted splice-correction therapy for neuromuscular disease.

Wood MJ, Gait MJ, Yin H.

Brain. 2010 Apr;133(Pt 4):957-72. doi: 10.1093/brain/awq002. Epub 2010 Feb 11. Review.

PMID:
20150322
[PubMed - indexed for MEDLINE]
Free Article
16.

Splicing, cis genetic variation and disease.

Jensen CJ, Oldfield BJ, Rubio JP.

Biochem Soc Trans. 2009 Dec;37(Pt 6):1311-5. doi: 10.1042/BST0371311. Review.

PMID:
19909267
[PubMed - indexed for MEDLINE]
17.

Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study.

Kinali M, Arechavala-Gomeza V, Feng L, Cirak S, Hunt D, Adkin C, Guglieri M, Ashton E, Abbs S, Nihoyannopoulos P, Garralda ME, Rutherford M, McCulley C, Popplewell L, Graham IR, Dickson G, Wood MJ, Wells DJ, Wilton SD, Kole R, Straub V, Bushby K, Sewry C, Morgan JE, Muntoni F.

Lancet Neurol. 2009 Oct;8(10):918-28. doi: 10.1016/S1474-4422(09)70211-X. Epub 2009 Aug 25. Erratum in: Lancet Neurol. 2009 Dec;8(12):1083.

PMID:
19713152
[PubMed - indexed for MEDLINE]
Free PMC Article
18.

Control of alternative splicing through siRNA-mediated transcriptional gene silencing.

Alló M, Buggiano V, Fededa JP, Petrillo E, Schor I, de la Mata M, Agirre E, Plass M, Eyras E, Elela SA, Klinck R, Chabot B, Kornblihtt AR.

Nat Struct Mol Biol. 2009 Jul;16(7):717-24. doi: 10.1038/nsmb.1620. Epub 2009 Jun 21.

PMID:
19543290
[PubMed - indexed for MEDLINE]
19.

Promoter-specific transcriptional interference and c-myc gene silencing by siRNAs in human cells.

Napoli S, Pastori C, Magistri M, Carbone GM, Catapano CV.

EMBO J. 2009 Jun 17;28(12):1708-19. doi: 10.1038/emboj.2009.139. Epub 2009 May 21.

PMID:
19461583
[PubMed - indexed for MEDLINE]
Free PMC Article
20.

Importin 8 is a gene silencing factor that targets argonaute proteins to distinct mRNAs.

Weinmann L, Höck J, Ivacevic T, Ohrt T, Mütze J, Schwille P, Kremmer E, Benes V, Urlaub H, Meister G.

Cell. 2009 Feb 6;136(3):496-507. doi: 10.1016/j.cell.2008.12.023. Epub 2009 Jan 22.

PMID:
19167051
[PubMed - indexed for MEDLINE]
Free Article

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