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Updated: Oct 11, 2025

Chemogenetic Regulation in Reprogrammed Stem Cell-derived Precursor Cells in Treating Neurodegenerative Diseases
Published on: May 2, 2025
Chemogenetics as a neuromodulatory approach to treating neuropsychiatric diseases and disorders
Jingwei Song1, Ruchit V Patel2, Massoud Sharif3
1National Institute on Drug Abuse Intramural Research Program, National Institutes of Health, Baltimore, MD 21224, USA; Department of Neuroscience, Johns Hopkins University, Baltimore, MD 21218, USA.
Abstract:
Chemogenetics enables precise, non-invasive, and reversible modulation of neural activity via the activation of engineered receptors that are pharmacologically selective to endogenous or exogenous ligands. With recent advances in therapeutic gene delivery, chemogenetics is poised to support novel interventions against neuropsychiatric diseases and disorders. To evaluate its translational potential, we performed a scoping review of applications of chemogenetics that led to the reversal of molecular and behavioral deficits in studies relevant to neuropsychiatric diseases and disorders. In this review, we present these findings and discuss the potential and challenges for using chemogenetics as a precision medicine-based neuromodulation strategy.
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