Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse

[1]  T. Partridge,et al.  Microbubble ultrasound improves the efficiency of gene transduction in skeletal muscle in vivo with reduced tissue damage , 2003, Gene Therapy.

[2]  Q. Lu,et al.  Non-viral gene delivery in skeletal muscle: a protein factory , 2003, Gene Therapy.

[3]  S. Takeda,et al.  Adeno-associated virus vector-mediated gene transfer into dystrophin-deficient skeletal muscles evokes enhanced immune response against the transgene product , 2002, Gene Therapy.

[4]  Federica Gemignani,et al.  Systemically delivered antisense oligomers upregulate gene expression in mouse tissues , 2002, Nature Biotechnology.

[5]  C. Mann,et al.  Improved antisense oligonucleotide induced exon skipping in the mdx mouse model of muscular dystrophy , 2002, The journal of gene medicine.

[6]  G. Dickson,et al.  Screening for antisense modulation of dystrophin pre-mRNA splicing , 2002, Neuromuscular Disorders.

[7]  G. V. Ommen,et al.  Targeted exon skipping as a potential gene correction therapy for Duchenne muscular dystrophy , 2002, Neuromuscular Disorders.

[8]  Ying Tang,et al.  Adeno-associated virus vector-mediated minidystrophin gene therapy improves dystrophic muscle contractile function in mdx mice. , 2002, Human gene therapy.

[9]  I. Bozzoni,et al.  Chimeric snRNA molecules carrying antisense sequences against the splice junctions of exon 51 of the dystrophin pre-mRNA induce exon skipping and restoration of a dystrophin synthesis in Δ48-50 DMD cells , 2002, Proceedings of the National Academy of Sciences of the United States of America.

[10]  N A Curtin,et al.  Isometric and isovelocity contractile performance of red muscle fibres from the dogfish Scyliorhinus canicula. , 2002, The Journal of experimental biology.

[11]  J. Faulkner,et al.  Force and power output of fast and slow skeletal muscles from mdx mice 6‐28 months old , 2001, The Journal of physiology.

[12]  Leaf Huang,et al.  Transfer of full-length Dmd to the diaphragm muscle of Dmd(mdx/mdx) mice through systemic administration of plasmid DNA. , 2001, Molecular therapy : the journal of the American Society of Gene Therapy.

[13]  K. Fischbeck,et al.  Gentamicin treatment of Duchenne and Becker muscular dystrophy due to nonsense mutations , 2001, Annals of neurology.

[14]  C. Mann,et al.  Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse. , 2001, Proceedings of the National Academy of Sciences of the United States of America.

[15]  Juan Li,et al.  Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model. , 2000, Proceedings of the National Academy of Sciences of the United States of America.

[16]  T. Rando,et al.  Rescue of dystrophin expression in mdx mouse muscle by RNA/DNA oligonucleotides. , 2000, Proceedings of the National Academy of Sciences of the United States of America.

[17]  S. Wilton,et al.  Massive Idiosyncratic Exon Skipping Corrects the Nonsense Mutation in Dystrophic Mouse Muscle and Produces Functional Revertant Fibers by Clonal Expansion , 2000, The Journal of cell biology.

[18]  T. Partridge,et al.  Dynamics of Myoblast Transplantation Reveal a Discrete Minority of Precursors with Stem Cell–like Properties as the Myogenic Source , 1999, The Journal of cell biology.

[19]  Q. L. Lu,et al.  A New Blocking Method for Application of Murine Monoclonal Antibody to Mouse Tissue Sections , 1998, The journal of histochemistry and cytochemistry : official journal of the Histochemistry Society.

[20]  N. Laing,et al.  Revertant fibres: a possible genetic therapy for Duchenne muscular dystrophy? , 1997, Neuromuscular Disorders.

[21]  T. Vulliamy,et al.  Exon skipping and translation in patients with frameshift deletions in the dystrophin gene. , 1993, American journal of human genetics.

[22]  Z. Dominski,et al.  Restoration of correct splicing in thalassemic pre-mRNA by antisense oligonucleotides. , 1993, Proceedings of the National Academy of Sciences of the United States of America.

[23]  N. Laing Molecular genetics and genetic counselling for Duchenne/Becker muscular dystrophy. , 1993, Molecular and cell biology of human diseases series.

[24]  K. Davies,et al.  Very mild muscular dystrophy associated with the deletion of 46% of dystrophin , 1990, Nature.

[25]  A. Monaco,et al.  An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locus. , 1988, Genomics.

[26]  Eric P. Hoffman,et al.  Dystrophin: The protein product of the duchenne muscular dystrophy locus , 1987, Cell.