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Empirical Bayes accomodation of batch-effects in microarray data using identical replicate reference samples: application to RNA expression profiling of blood from Duchenne muscular dystrophy patients
Wynn L Walker et al.
BMC Genomics 9 (1), 494 (20 Oct 2008)
 
Octa-guanidine Morpholino Restores Dystrophin Expression in Cardiac and Skeletal Muscles and Ameliorates Pathology in Dystrophic mdx Mice
Bo Wu et al.
Mol Ther 17 (5), 864-71 (10 Mar 2009)
Vivo-Morpholinos for exon skipping in mice
 
Efficacy of systemic morpholino exon-skipping in duchenne dystrophy dogs.
Toshifumi Yokota et al.
Annals of neurology, (13 Mar 2009)
Exon-skipping induced by Morpholinos causes systemic expression of functional dystrophin in a DMD dog model.
Posted by jonmoulton to DMD morpholino Beagle on Fri Apr 03 2009 at 21:41 UTC | info | related
 
Gene knockdowns in adult animals: PPMOs and vivo-morpholinos.
Jon D Moulton and Shan Jiang
Molecules (Basel, Switzerland) 14 (3), 1304-23 (2009)
Review of some recent in vivo work with delivery-enabled Morpholino oligos. Open-access.
Posted by jonmoulton (who is an author) to Vivo-Morpholino PPMO DMD morpholino antiviral on Fri Apr 03 2009 at 21:38 UTC | info | related
 
Autologous transplantation of muscle-derived CD133+ stem cells in Duchenne muscle patients.
Y Torrente et al.
Cell transplantation 16 (6), 563-77 (2007)
Posted by SByer and 1 other to DMD CD133 effective Repair in muscle on Tue Feb 17 2009 at 11:24 UTC | info | related
 
PLoS ONE: Correlation of Circulating CD133+ Progenitor Subclasses with a Mild Phenotype in Duchenne Muscular Dystrophy Patients
www.plosone.org
Posted by SByer and 1 other to CD133-Low DMD RE incidence on Tue Feb 17 2009 at 11:14 UTC | info | related
 
A renaissance for antisense oligonucleotide drugs in neurology: exon skipping breaks new ground.
Toshifumi Yokota et al.
Archives of neurology 66 (1), 32-8 (Jan 2009)
Yokota T, Takeda S, Lu QL, Partridge TA, Nakamura A, Hoffman EP. A renaissance for antisense oligonucleotide drugs in neurology: exon skipping breaks new ground. Arch Neurol. 2009 Jan;66(1):32-8.
 
In vivo comparison of 2'-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping.
Hans A Heemskerk et al.
The journal of gene medicine, (12 Jan 2009)
Heemskerk HA, de Winter CL, de Kimpe SJ, van Kuik-Romeijn P, Heuvelmans N, Platenburg GJ, van Ommen GJ, van Deutekom JC, Aartsma-Rus A. In vivo comparison of 2'-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping. J Gene Med. 2009 Jan 12. [Epub ahead of print]
 
Proteomic profiling of antisense-induced exon skipping reveals reversal of pathobiochemical abnormalities in dystrophic mdx diaphragm.
Philip Doran et al.
Proteomics, (08 Jan 2009)
Doran P, Wilton SD, Fletcher S, Ohlendieck K. Proteomic profiling of antisense-induced exon skipping reveals reversal of pathobiochemical abnormalities in dystrophic mdx diaphragm. Proteomics. 2009 Jan 8. [Epub ahead of print]
Posted by jonmoulton to DMD morpholino MDX duchenne on Mon Jan 12 2009 at 20:18 UTC | info | related
 
Therapeutic Potential of Splice-Switching Oligonucleotides
Bauman J, Jearawiriyapaisarn N, and Kole R
Oligonucleotides
Bauman J, Jearawiriyapaisarn N, Kole R. Therapeutic Potential of Splice-Switching Oligonucleotides. Oligonucleotides. 2009 Jan 6. [Epub ahead of print]

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