Repository logo
 

A humanized chemogenetic system inhibits murine pain-related behavior and hyperactivity in human sensory neurons.

Accepted version
Peer-reviewed

Type

Article

Change log

Authors

Perez-Sanchez, Jimena  ORCID logo  https://orcid.org/0000-0002-4967-5418
Middleton, Steven J  ORCID logo  https://orcid.org/0000-0002-0708-7335
Pattison, Luke A 
Hilton, Helen 
Ali Awadelkareem, Mosab  ORCID logo  https://orcid.org/0000-0001-6898-6310

Abstract

Hyperexcitability in sensory neurons is known to underlie many of the maladaptive changes associated with persistent pain. Chemogenetics has shown promise as a means to suppress such excitability, yet chemogenetic approaches suitable for human applications are needed. PSAM4-GlyR is a modular system based on the human α7 nicotinic acetylcholine and glycine receptors, which responds to inert chemical ligands and the clinically approved drug varenicline. Here, we demonstrated the efficacy of this channel in silencing both mouse and human sensory neurons by the activation of large shunting conductances after agonist administration. Virally mediated expression of PSAM4-GlyR in mouse sensory neurons produced behavioral hyposensitivity upon agonist administration, which was recovered upon agonist washout. Stable expression of the channel led to similar reversible suppression of pain-related behavior even after 10 months of viral delivery. Mechanical and spontaneous pain readouts were also ameliorated by PSAM4-GlyR activation in acute and joint pain inflammation mouse models. Furthermore, suppression of mechanical hypersensitivity generated by a spared nerve injury model of neuropathic pain was also observed upon activation of the channel. Effective silencing of behavioral hypersensitivity was reproduced in a human model of hyperexcitability and clinical pain: PSAM4-GlyR activation decreased the excitability of human-induced pluripotent stem cell-derived sensory neurons and spontaneous activity due to a gain-of-function NaV1.7 mutation causing inherited erythromelalgia. Our results demonstrate the contribution of sensory neuron hyperexcitability to neuropathic pain and the translational potential of an effective, stable, and reversible humanized chemogenetic system for the treatment of pain.

Description

Keywords

Humans, Mice, Animals, Neuralgia, Sensory Receptor Cells, Mutation, Ganglia, Spinal

Journal Title

Sci Transl Med

Conference Name

Journal ISSN

1946-6234
1946-6242

Volume Title

15

Publisher

American Association for the Advancement of Science (AAAS)
Sponsorship
MRC (MR/W002426/1)
BBSRC (BB/V509528/1)