Development of antisense oligonucleotides to down-regulate alpha-synuclein 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 characterization, and solution-phase conjugation of PNAs to cell-penetrating peptides. In vitro characterisation of PNAs using neuroblastoma cell line, Protein extraction and western blotting.
In this project, we aim to 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.
Take part in this project
Students who are applying to study at The Florey can register their interest in this project. Refer to our step-by-step guide to help you with your application.