Research Topics
| Toshifumi YokotaSummaryAffiliation: Children's National Medical Center Country: USA Publications
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Detail Information
Publications
Potential of oligonucleotide-mediated exon-skipping therapy for Duchenne muscular dystrophyToshifumi Yokota
Children s National Medical Center, Research Center for Genetic Medicine, Washington, DC 20010, USA
Expert Opin Biol Ther 7:831-42. 2007..This review summarizes recent progress in exon-skipping therapy and discusses future perspectives with regard to human clinical trials...
Optimizing exon skipping therapies for DMDT Yokota
Children s National Medical Center, Center for Genetic Medicine, Washington, DC 20010, USA
Acta Myol 26:179-84. 2007..This review summarizes recent progress in exon skipping therapy and discusses future strategies...
A renaissance for antisense oligonucleotide drugs in neurology: exon skipping breaks new groundToshifumi Yokota
Research Center for Genetic Medicine, Children s National Medical Center, 111 Michigan Ave NW, Washington, DC 20010, USA
Arch Neurol 66:32-8. 2009....
Antisense oligo-mediated multiple exon skipping in a dog model of duchenne muscular dystrophyToshifumi Yokota
Research Center for Genetic Medicine, Children s National Medical Center, 111 Michigan Avenue, NW, Washington, DC, USA
Methods Mol Biol 709:299-312. 2011....
Restoring dystrophin expression in duchenne muscular dystrophy muscle progress in exon skipping and stop codon read throughEric P Hoffman
Research Center for Genetic Medicine, Children s National Medical Center, 111 Michigan Ave NW, Washington, DC 20010, USA
Am J Pathol 179:12-22. 2011..We discuss the status of these two methods aimed at inducing de novo dystrophin production from mutant genes and review implications for other disorders...
Efficacy of systemic morpholino exon-skipping in Duchenne dystrophy dogsToshifumi Yokota
Research Center for Genetic Medicine, Children s National Medical Center, Washington, DC 20010, USA
Ann Neurol 65:667-76. 2009..We sought to test efficacy and toxicity of intravenous oligonucleotide (morpholino)-induced exon skipping in the DMD dog model...
Expansion of revertant fibers in dystrophic mdx muscles reflects activity of muscle precursor cells and serves as an index of muscle regenerationToshifumi Yokota
Muscle Cell Biology Group, Medical Research Council Clinical Science Centre, Hammersmith Hospital Campus, Imperial College School of Medicine, London University, Du Cane Road, London, W12 0NN, UK
J Cell Sci 119:2679-87. 2006..This expansion of revertant clusters depicts the cumulative history of regeneration, thus providing a useful index for functional evaluation of therapies that counteract muscle degeneration...
Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal musclesQi Long Lu
Muscle Cell Biology, Medical Research Council Clinical Science Centre, Hammersmith Hospital, Du Cane Road, London W12 0NN, UK
Proc Natl Acad Sci U S A 102:198-203. 2005..We conclude that a significant therapeutic effect may be achieved by further optimization in dose and regime of administration of antisense oligonucleotide...
Micro-dystrophin cDNA ameliorates dystrophic phenotypes when introduced into mdx mice as a transgeneMiki Sakamoto
Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, 4 1 1 Ogawa Higashi, Kodaira, Tokyo 187 8502, Japan
Biochem Biophys Res Commun 293:1265-72. 2002..These data suggest that the rod structure, and its length in particular, is crucial for the function of micro-dystrophin...
Alpha1-syntrophin-deficient skeletal muscle exhibits hypertrophy and aberrant formation of neuromuscular junctions during regenerationYukio Hosaka
Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, 4 1 1 Ogawa Higashi, Kodaira 187 8502, Tokyo, Japan
J Cell Biol 158:1097-107. 2002....
