Efficacy of peptide nucleic acid and selected conjugates against specific cellular pathologies of amyotrophic lateral sclerosis

Elisse C. Browne, Sonam Parakh, Luke F. Duncan, Steven J. Langford, Julie D Atkin, Belinda M. Abbott

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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.

Original languageEnglish
Pages (from-to)1520-1527
Number of pages8
JournalBioorganic & Medicinal Chemistry
Volume24
Issue number7
DOIs
Publication statusPublished - 1 Apr 2016

Keywords

  • Amyotrophic lateral sclerosis
  • Peptide nucleic acid
  • Vitamin conjugation

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