Toshifumi Yokota

Micro-dystrophin cDNA ameliorates dystrophic phenotypes when introduced into mdx mice as a transgene.

Alpha1-syntrophin-deficient skeletal muscle exhibits hypertrophy and aberrant formation of neuromuscular junctions during regeneration.

Alpha1-syntrophin modulates turnover of ABCA1.

Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles.

Expansion of revertant fibers in dystrophic mdx muscles reflects activity of muscle precursor cells and serves as an index of muscle regeneration.

Potential of oligonucleotide-mediated exon-skipping therapy for Duchenne muscular dystrophy.

A renaissance for antisense oligonucleotide drugs in neurology: exon skipping breaks new ground.

Efficacy of systemic morpholino exon-skipping in Duchenne dystrophy dogs.

Antisense PMO found in dystrophic dog model was effective in cells from exon 7-deleted DMD patient.

In-frame dystrophin following exon 51-skipping improves muscle pathology and function in the exon 52-deficient mdx mouse.

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

Antisense oligo-mediated multiple exon skipping in a dog model of duchenne muscular dystrophy.

Restoring dystrophin expression in duchenne muscular dystrophy muscle progress in exon skipping and stop codon read through.

Pathogenic exon-trapping by SVA retrotransposon and rescue in Fukuyama muscular dystrophy.

Exon skipping for nonsense mutations in Duchenne muscular dystrophy: too many mutations, too few patients?

Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery.

Extensive and prolonged restoration of dystrophin expression with vivo-morpholino-mediated multiple exon skipping in dystrophic dogs.

Highly efficient in vivo delivery of PMO into regenerating myotubes and rescue in laminin-α2 chain-null congenital muscular dystrophy mice.

Mutation types and aging differently affect revertant fiber expansion in dystrophic mdx and mdx52 mice.

Development of multiexon skipping antisense oligonucleotide therapy for Duchenne muscular dystrophy.

α1-Syntrophin-deficient mice exhibit impaired muscle force recovery after osmotic shock.

Morpholino treatment improves muscle function and pathology of Pitx1 transgenic mice.

Skipping multiple exons of dystrophin transcripts using cocktail antisense oligonucleotides.

New developments in exon skipping and splice modulation therapies for neuromuscular diseases.

Antisense therapy in neurology.

Long-term efficacy of systemic multiexon skipping targeting dystrophin exons 45-55 with a cocktail of vivo-morpholinos in mdx52 mice.

In silico screening based on predictive algorithms as a design tool for exon skipping oligonucleotides in Duchenne muscular dystrophy.

Nonmechanical Roles of Dystrophin and Associated Proteins in Exercise, Neuromuscular Junctions, and Brains.

Muscular Dystrophy: Disease Mechanisms and Therapies.

Dystrophin-deficient large animal models: translational research and exon skipping.

Antisense oligonucleotide drugs for Duchenne muscular dystrophy: how far have we come and what does the future hold?

Deletion of exons 3-9 encompassing a mutational hot spot in the DMD gene presents an asymptomatic phenotype, indicating a target region for multiexon skipping therapy.

Current Translational Research and Murine Models For Duchenne Muscular Dystrophy.

Impaired regenerative capacity and lower revertant fibre expansion in dystrophin-deficient mdx muscles on DBA/2 background.

Systemic Delivery of Morpholinos to Skip Multiple Exons in a Dog Model of Duchenne Muscular Dystrophy.

Effects of systemic multiexon skipping with peptide-conjugated morpholinos in the heart of a dog model of Duchenne muscular dystrophy.

LNA/DNA mixmer-based antisense oligonucleotides correct alternative splicing of the SMN2 gene and restore SMN protein expression in type 1 SMA fibroblasts.

Quantitative Antisense Screening and Optimization for Exon 51 Skipping in Duchenne Muscular Dystrophy.

Immortalized Muscle Cell Model to Test the Exon Skipping Efficacy for Duchenne Muscular Dystrophy.

Exon Skipping Therapy Using Phosphorodiamidate Morpholino Oligomers in the mdx52 Mouse Model of Duchenne Muscular Dystrophy.

Designing Effective Antisense Oligonucleotides for Exon Skipping.

Skipping Multiple Exons to Treat DMD-Promises and Challenges.