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Maleeva, Galyna ; Nin-Hill, Alba ; Wirth, Ulrike ; Rustler, Karin ; Ranucci, Matteo ; Opar, Ekin ; Rovira, Carme ; Bregestovski, Piotr ; Zeilhofer, Hanns Ulrich ; König, Burkhard ; Alfonso-Prieto, Mercedes ; Gorostiza, Pau

Light-Activated Agonist-Potentiator of GABAA Receptors for Reversible Neuroinhibition in Wildtype Mice

Maleeva, Galyna , Nin-Hill, Alba , Wirth, Ulrike, Rustler, Karin , Ranucci, Matteo , Opar, Ekin, Rovira, Carme , Bregestovski, Piotr , Zeilhofer, Hanns Ulrich , König, Burkhard , Alfonso-Prieto, Mercedes and Gorostiza, Pau (2024) Light-Activated Agonist-Potentiator of GABAA Receptors for Reversible Neuroinhibition in Wildtype Mice. Journal of the American Chemical Society 146 (42), pp. 28822-28831.

Date of publication of this fulltext: 23 Oct 2024 12:44
Article
DOI to cite this document: 10.5283/epub.59429


Abstract

Gamma aminobutyric acid type A receptors (GABAARs) play a key role in the mammalian central nervous system (CNS) as drivers of neuroinhibitory circuits, which are commonly targeted for therapeutic purposes with potentiator drugs. However, due to their widespread expression and strong inhibitory action, systemic pharmaceutical potentiation of GABAARs inevitably causes adverse effects regardless of ...

Gamma aminobutyric acid type A receptors (GABAARs) play a key role in the mammalian central nervous system (CNS) as drivers of neuroinhibitory circuits, which are commonly targeted for therapeutic purposes with potentiator drugs. However, due to their widespread expression and strong inhibitory action, systemic pharmaceutical potentiation of GABAARs inevitably causes adverse effects regardless of the drug selectivity. Therefore, therapeutic guidelines must often limit or exclude clinically available GABAAR potentiators, despite their high efficacy, good biodistribution, and favorable molecular properties. One solution to this problem is to use drugs with light-dependent activity (photopharmacology) in combination with on-demand, localized illumination. However, a suitable light-activated potentiator of GABAARs has been elusive so far for use in wildtype mammals. We have met this need by developing azocarnil, a diffusible GABAergic agonist-potentiator based on the anxiolytic drug abecarnil that is inactive in the dark and activated by visible violet light. Azocarnil can be rapidly deactivated with green light and by thermal relaxation in the dark. We demonstrate that it selectively inhibits neuronal currents in hippocampal neurons in vitro and in the dorsal horns of the spinal cord of mice, decreasing the mechanical sensitivity as a function of illumination without displaying systemic adverse effects. Azocarnil expands the in vivo photopharmacological toolkit with a novel chemical scaffold and achieves a milestone toward future phototherapeutic applications to safely treat muscle spasms, pain, anxiety, sleep disorders, and epilepsy.



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Details

Item typeArticle
Journal or Publication TitleJournal of the American Chemical Society
Publisher:American Chemical Society
Volume:146
Number of Issue or Book Chapter:42
Page Range:pp. 28822-28831
Date9 October 2024
InstitutionsChemistry and Pharmacy > Institut für Organische Chemie > Lehrstuhl Prof. Dr. Burkhard König
Identification Number
ValueType
10.1021/jacs.4c08446DOI
KeywordsElectromagnetic radiation; Light Neurophysiology; Pharmaceuticals; Receptors
Dewey Decimal Classification500 Science > 540 Chemistry & allied sciences
StatusPublished
RefereedYes, this version has been refereed
Created at the University of RegensburgPartially
URN of the UB Regensburgurn:nbn:de:bvb:355-epub-594298
Item ID59429

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