Huntington’s disease

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ABC transporters play an important role in different neurodegenerative diseases. We therefore investigate also Huntington’s disease as a genetic disease with known disease locus. To assess effects of the CAG repeat expansions in the Huntingtin gene, we generated a new model zQ175dn based on an available model (see below). Currently, we investigate a new treatment based on our recent results an a re-purposing strategy. This treatment shows efficient disease modifying effects.
Additionally, we are insterested to assess ABCC1 and ABCA7 function for the disease initiation and disease progression. Recent results show that an important common modulator may be responsible for disease modification in the mouse model and is regulated via ABCA7.

* Project Links:

More about… ABCHD
More about… A7HD application to JPND 2024….
More about… HDFTY repurposing study planned to start 2024 ….

* Published Research Papers (selection):

  • HD_BPMDS—a curated binary pattern multitarget dataset of Huntington’s disease–targeting agents
    J Cheminformatics (2023)

    Namasivayam V, Pahnke J, Stefan SM

    15(1):109 PubMed PDF
    Open Data Access

  • A novel Huntington’s disease assessment platform to support future drug discovery and development
    Int J of Molecular Sciences (2022)

    Wu J, Möhle L, Brüning T, Eiriz I, Rafehi M, Stefan K, Stefan SM, and Pahnke J
    23(23):14763 PubMed PDF
    Open Data Access