title: Myotonic dystrophy: antisense oligonucleotide inhibition of DMPK gene expression in vitro.
abstract: Antisense phosphorothioate oligonucleotides, targeted against the first codon starting region of DMPK mRNA, were successfully used in K562 and HepG2 cells to decrease DMPK expression. The most effective antisense oligo, MIO1, when added to K562 cells, shows a 75% reduction of the DMPK gene expression 6 hours after addition. The same molecule, when encapsulated in liposomes, delays myotonin mRNA decrease at 24 hours after cell treatment. This considerable success with such inhibition in vitro could be utilised to generate a cell model to study myotonic dystrophy (DM) chemio-physiological alterations.
authors.affiliation: Istituto di Biochimica delle Macromolecole, 2.a Università di Napoli, Facoltà di Medicina, Italy.
authors.forename: U
authors.initials: U
authors.lastname: Galderisi
authors.forename: M
authors.initials: M
authors.lastname: Cipollaro
authors.forename: M A
authors.initials: MA
authors.lastname: Melone
authors.forename: G
authors.initials: G
authors.lastname: Iacomino
authors.forename: G
authors.initials: G
authors.lastname: Di Bernardo
authors.forename: G
authors.initials: G
authors.lastname: Galano
authors.forename: R
authors.initials: R
authors.lastname: Contrufo
authors.forename: V
authors.initials: V
authors.lastname: Zappia
authors.forename: A
authors.initials: A
authors.lastname: Cascino
mesh_terms: D001483:Base Sequence; D002460:Cell Line; D005786:Gene Expression Regulation / Q000187:drug effects*; D006801:Humans; D008969:Molecular Sequence Data; D009223:Myotonic Dystrophy / Q000235:genetics*; D065636:Myotonin-Protein Kinase; D016376:Oligonucleotides, Antisense / Q000494:pharmacology*; D017346:Protein Serine-Threonine Kinases / Q000235:genetics*; D012333:RNA, Messenger / Q000235:genetics; D013873:Thionucleotides / Q000494:pharmacology*
chemical_list: C585517:DMPK protein, human; D016376:Oligonucleotides, Antisense; D012333:RNA, Messenger; D013873:Thionucleotides; D065636:Myotonin-Protein Kinase; D017346:Protein Serine-Threonine Kinases