title: Efficacy of peptide nucleic acid and selected conjugates against specific cellular pathologies of amyotrophic lateral sclerosis.
abstract: Cellular studies have been undertaken on a nonamer peptide nucleic acid (PNA) sequence, which binds to mRNA encoding superoxide dismutase 1, and a series of peptide nucleic acids conjugated to synthetic lipophilic vitamin analogs including a recently prepared menadione (vitamin K) analog. Reduction of both mutant superoxide dismutase 1 inclusion formation and endoplasmic reticulum stress, two of the key cellular pathological hallmarks in amyotrophic lateral sclerosis, by two of the prepared PNA oligomers is reported for the first time.
authors.affiliation: Department of Chemistry and Physics, La Trobe Institute for Molecular Science, La Trobe University, Melbourne 3086, Australia.
authors.forename: Elisse C
authors.initials: EC
authors.lastname: Browne
authors.affiliation: Department of Biochemistry and Genetics, La Trobe Institute for Molecular Science, La Trobe University, Melbourne 3086, Australia; Department of Biomedical Sciences, Faculty of Medicine and Health Science, Macquarie University, Sydney 2109, Australia.
authors.forename: Sonam
authors.initials: S
authors.lastname: Parakh
authors.affiliation: Department of Chemistry and Physics, La Trobe Institute for Molecular Science, La Trobe University, Melbourne 3086, Australia.
authors.forename: Luke F
authors.initials: LF
authors.lastname: Duncan
authors.affiliation: School of Chemistry, Monash University, Clayton 3800, Australia.
authors.forename: Steven J
authors.initials: SJ
authors.lastname: Langford
authors.affiliation: Department of Biochemistry and Genetics, La Trobe Institute for Molecular Science, La Trobe University, Melbourne 3086, Australia; Department of Biomedical Sciences, Faculty of Medicine and Health Science, Macquarie University, Sydney 2109, Australia.
authors.forename: Julie D
authors.initials: JD
authors.lastname: Atkin
authors.affiliation: Department of Chemistry and Physics, La Trobe Institute for Molecular Science, La Trobe University, Melbourne 3086, Australia. Electronic address: b.abbott@latrobe.edu.au.
authors.forename: Belinda M
authors.initials: BM
authors.lastname: Abbott
mesh_terms: D000690:Amyotrophic Lateral Sclerosis / Q000188:drug therapy* / Q000235:genetics / Q000378:metabolism / Q000473:pathology*; D000818:Animals; D002478:Cells, Cultured; D059865:Endoplasmic Reticulum Stress / Q000187:drug effects*; D051379:Mice; D020135:Peptide Nucleic Acids / Q000138:chemical synthesis / Q000737:chemistry / Q000494:pharmacology*; D013482:Superoxide Dismutase / Q000172:deficiency / Q000235:genetics / Q000378:metabolism*; D000072105:Superoxide Dismutase-1; D013816:Thermodynamics; D014815:Vitamins / Q000737:chemistry / Q000494:pharmacology*
chemical_list: D020135:Peptide Nucleic Acids; D014815:Vitamins; D013482:Superoxide Dismutase; D000072105:Superoxide Dismutase-1
keywords: Amyotrophic lateral sclerosis; Peptide nucleic acid; Vitamin conjugation