Induction of Dystrophin Expression by Exon Skipping in mdx Mice Following Intramuscular Injection of Antisense Oligonucleotides Complexed with PEG

Induction of Dystrophin Expression by Exon Skipping in mdx Mice Following Intramuscular Injection of Antisense Oligonucleotides Complexed with PEG–PEI Copolymers

Published:2006-07 Issue:1 Volume:14 Page:88-96
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Williams Jason H.,Sirsi Shashank R.,Latta Daniel R.,Lutz Gordon J.

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference46 articles.

1. Targeted exon skipping as a potential gene correction therapy for Duchenne muscular dystrophy;Aartsma-Rus;Neuromuscul. Disord.,2002

2. Therapeutic antisense-induced exon skipping in cultured muscle cells from six different DMD patients;Aartsma-Rus;Hum. Mol. Genet.,2003

3. Antisense-induced multiexon skipping for Duchenne muscular dystrophy makes more sense;Aartsma-Rus;Am. J. Hum. Genet.,2004

4. Intracellular route and transcriptional competence of polyethylenimine–DNA complexes;Bieber;J. Controlled Release,2002

5. A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine;Boussif;Proc. Natl. Acad. Sci. USA,1995

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