Novel pharmacotherapies for the treatment of central TH deprivation associated with Mct8/Oatp1c1 deficiency (P05)

• Subject Area: Endocrinology, Diabetology, Metabolism
• Term: since 2020
• Project identifier: Deutsche Forschungsgemeinschaft (DFG) - Project number 424957847

In this proposal we will evaluate a series of novel TH/peptide chimeras for their ability to improve brain TH deficiency associated with AHDS. Studies will include LSFM and MS-based assessment of drug BBB permeability, advanced in vitro and in vivo approaches to delineate proof-of-concept and drugability of central Mct8 deficiency and state-of-the-art genetic, microscopic, transcriptomic and proteomic approaches to further advance our knowledge on the neuronal and behavioral underpinnings underlying disturbed Mct8 expression and function.

• DFG Programme: CRC/Transregios
• Subproject of: TRR 296: Local control of TH action (LocoTact)
• Applicant Institution: Universität Duisburg-Essen
• Project Heads: Privatdozent Dr. Timo Müller; Professor Dr. Paul Pfluger