Tropane-Based Ibogaine Analog Rescues Folding-Deficient Serotonin and Dopamine Transporters

Bhat, Shreyas, Guthrie, Daryl A., Kasture, Ameya, El-Kasaby, Ali, Cao, Jianjing, Bonifazi, Alessandro, Ku, Therese, Giancola, JoLynn B., Hummel, Thomas, Freissmuth, Michael, Newman, Amy Hauck · 2020 · ACS Pharmacology & Translational Science

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Tropane-based ibogaine analogue (9b) rescued 6/13 disease-associated DAT mutants causing infantile Parkinsonism/dystonia. Novel pharmacochaperone lead for DTDS with improved therapeutic index.

Dosing: 0.06–30 µM (in vitro); 10–100 µM (Drosophila feeding) (not-applicable)