Induction of dystrophin expression by exon skipping in mdx mice following intramuscular injection of antisense oligonucleotides complexed with PEG-PEI copolymers (Q82604893)

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scientific article published on 20 February 2006

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English	Induction of dystrophin expression by exon skipping in mdx mice following intramuscular injection of antisense oligonucleotides complexed with PEG-PEI copolymers	scientific article published on 20 February 2006

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scholarly article

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17 January 2020

Induction of dystrophin expression by exon skipping in mdx mice following intramuscular injection of antisense oligonucleotides complexed with PEG-PEI copolymers (English)

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17 January 2020

author name string

Jason H Williams

1

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17 January 2020

Shashank R Sirsi

2

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17 January 2020

Daniel R Latta

3

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17 January 2020

Gordon J Lutz

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20 February 2006

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Molecular Therapy

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1

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88-96

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17 January 2020

Targeted exon skipping as a potential gene correction therapy for Duchenne muscular dystrophy.

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Therapeutic antisense-induced exon skipping in cultured muscle cells from six different DMD patients.

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Antisense-induced multiexon skipping for Duchenne muscular dystrophy makes more sense

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Intracellular route and transcriptional competence of polyethylenimine-DNA complexes

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A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine

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Targeted exon skipping in transgenic hDMD mice: A model for direct preclinical screening of human-specific antisense oligonucleotides

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Physicochemical and biological characterization of polyethylenimine-graft-poly(ethylene glycol) block copolymers as a delivery system for oligonucleotides and ribozymes

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Modification of splicing in the dystrophin gene in cultured Mdx muscle cells by antisense oligoribonucleotides

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Terminal antisense oligonucleotide modifications can enhance induced exon skipping

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Physiochemical properties of low and high molecular weight poly(ethylene glycol)-grafted poly(ethylene imine) copolymers and their complexes with oligonucleotides

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Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping

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The cellular delivery of antisense oligonucleotides and ribozymes

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A new antisense oligonucleotide delivery system based on self-assembled ODN-PEG hybrid conjugate micelles

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Gene transfer with modified polyethylenimines

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The structure of PEG-modified poly(ethylene imines) influences biodistribution and pharmacokinetics of their complexes with NF-kappaB decoy in mice

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Non-viral gene delivery in skeletal muscle: a protein factory

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Microbubble ultrasound improves the efficiency of gene transduction in skeletal muscle in vivo with reduced tissue damage

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

1 reference

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

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Improved antisense oligonucleotide induced exon skipping in the mdx mouse model of muscular dystrophy

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Prospects for cationic polymers in gene and oligonucleotide therapy against cancer

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Polyethylenimine-graft-poly(ethylene glycol) copolymers: influence of copolymer block structure on DNA complexation and biological activities as gene delivery system

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Expression of full-length and truncated dystrophin mini-genes in transgenic mdx mice

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Engineering synthetic vectors for improved DNA delivery: insights from intracellular pathways

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Cancer siRNA therapy by tumor selective delivery with ligand-targeted sterically stabilized nanoparticle

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Effective intracellular delivery of oligonucleotides in order to make sense of antisense

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Repeated intrathecal administration of plasmid DNA complexed with polyethylene glycol-grafted polyethylenimine led to prolonged transgene expression in the spinal cord

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Efficient active transport of gene nanocarriers to the cell nucleus

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Effect of polyethylene glycol on gene delivery of polyethylenimine

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Electrophoretic separation of rat skeletal muscle myosin heavy-chain isoforms.

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Non-viral gene therapy: polycation-mediated DNA delivery

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7 January 2021

Antisense-induced exon skipping restores dystrophin expression in DMD patient derived muscle cells

1 reference

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7 January 2021

Advances in Duchenne muscular dystrophy gene therapy

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Polyion complex micelles with protein-modified corona for receptor-mediated delivery of oligonucleotides into cells.

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Nanogels for oligonucleotide delivery to the brain

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Self-assembly of polyamine-poly(ethylene glycol) copolymers with phosphorothioate oligonucleotides.

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Expression of human full-length and minidystrophin in transgenic mdx mice: implications for gene therapy of Duchenne muscular dystrophy

1 reference

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Enhanced in vivo delivery of antisense oligonucleotides to restore dystrophin expression in adult mdx mouse muscle

1 reference

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Barriers to nonviral gene delivery

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Antisense oligonucleotides in the treatment of Duchenne muscular dystrophy: Where are we now?

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Identifiers

10.1016/J.YMTHE.2005.11.025

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Europe PubMed Central

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:16488666%20AND%20SRC:MED&resulttype=core&format=json

17 January 2020

1 reference

Europe PubMed Central

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:16488666%20AND%20SRC:MED&resulttype=core&format=json

17 January 2020

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