Cross‐species systems biology target prioritization leads to the development of antisense oligonucleotides as a potential therapeutic approach for progressive supranuclear palsy
We developed a systems biology pipeline and nominated STOM, KANK2, and DDR2 as candidate gene targets for PSP. We identified ASOs that modulate these targets' expression without cellular toxicity, suggesting they may be suitable as potential therapeutic candidates. Importantly, the shared pathophysiology and molecular aberrations between PSP and other tauopathies, such as Alzheimer's disease, suggests that such therapeutic candidates may be repurposed for multiple neurodegenerative diseases, further accelerating, and streamlining the therapeutic pipeline.
Authors
- Adriana O. Mitchell
- Dennis W. Dickson (ORCID: https://orcid.org/0000-0001-7189-7917)
- Keiji Kawatani (ORCID: https://orcid.org/0009-0004-9180-3219)
- Joseph S. Reddy (ORCID: https://orcid.org/0000-0002-1783-4453)
- Takahisa Kanekiyo (ORCID: https://orcid.org/0000-0001-6751-9374)
- Özkan İş (ORCID: https://orcid.org/0000-0003-4070-6756)
- Yuhao Min (ORCID: https://orcid.org/0000-0001-8775-8743)
- Yasuteru Inoue (ORCID: https://orcid.org/0000-0003-4180-5908)
- Nilüfer Ertekin‐Taner (ORCID: https://orcid.org/0000-0003-4436-8889)
- Mariet Allen (ORCID: https://orcid.org/0000-0002-2685-427X)
- Zachary Quicksall (ORCID: https://orcid.org/0000-0002-8791-0925)
- Katie D Sotelo
- Xue Wang (ORCID: https://orcid.org/0000-0002-0423-1952)
- Thuy Nguyen (ORCID: https://orcid.org/0000-0003-2971-4077)
- Jeremiah Bergman
Institutions
- WinnMed (US)
- Mayo Clinic in Florida (US)
Publication Details
- Journal
- Alzheimer s & Dementia
- Published
- 2024-12-01
- DOI
- https://doi.org/10.1002/alz.090392
- Primary Topic
- Advanced biosensing and bioanalysis techniques
- Type
- article
- Field-Weighted Citation Impact
- 0.00