Development of antisense oligonucleotides as a potential treatment of Parkinson’s disease

Every year thousands of people are diagnosed with Parkinson’s disease (PD) with no known cure. Alpha-synuclein is one of the most compelling targets for new therapies in PD, with converging lines of evidence implicating a causative role in the disease and its progression through a toxic gain-of-function mechanism. Down-regulation of alpha-synuclein by with antisense oligonucleotides (ASOs) offer a new therapeutic modality for treating PD. ASOs is an attractive therapeutic strategy since it is agnostic to the molecular species or mechanism by which alpha-synuclein mediates neurotoxicity, both of which are unclear.

Skill acquisition: A broad range of skills will be acquired. Students will be trained on solid-phase PNA synthesis, HPLC purification, mass spectrometry characterisation, and solution-phase conjugation of PNAs to cell-penetrating peptides. In vitro characterisation of PNAs using neuroblastoma cell line, Protein extraction and western blotting.

Aim

  • Design and synthesise a novel class of ASO based on peptide nucleic acid (PNA) to block alpha-synuclein mRNA and reduce the level of alpha-synuclein.

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