Volume : 12, Issue : 06, June – 2025
Title:
GENE THERAPY APPROACHES IN ANXIETY AND EPILEPSY: TARGETING HTR2A AND NEUROPEPTIDE Y
Authors :
M. Ganga Raju, Mohammed Abdul Rafay, N.V.L Suvarchala Reddy, E. Ananya,B. Sindhura
Abstract :
Anxiety and epilepsy are complex neuropsychiatric disorders characterized by disrupted neural signaling and imbalances in neurotransmitter regulation. Conventional pharmacological treatments such as benzodiazepines, SSRIs, and antiepileptic drugs provide symptomatic relief but are often limited by side effects, drug resistance, and incomplete efficacy. These challenges have driven the exploration of more precise and long-lasting therapeutic alternatives, including gene therapy. Recent advances in molecular neuroscience have enabled targeted interventions at the genetic level, offering new hope for patients unresponsive to traditional therapies. This review focuses on gene therapy strategies targeting two key molecular regulators: the HTR2A gene, which encodes the 5-HT2A serotonin receptor implicated in anxiety modulation, and Neuropeptide Y (NPY), a neuropeptide known for its anticonvulsant and anxiolytic properties. By manipulating the expression or signaling pathways of these targets, researchers aim to restore neurochemical balance and improve neuronal resilience. Intranasal delivery methods using viral vectors, including adeno-associated virus (AAV), have shown promise in preclinical models, enabling non-invasive, brain-targeted gene delivery with minimal systemic toxicity.
We summarize the current state of gene therapy research in anxiety and epilepsy, highlighting preclinical successes, delivery systems, and safety considerations. The review also explores emerging tools such as CRISPR/Cas9, RNA interference (RNAi), and ligand-directed viral vectors that enhance specificity and efficacy. While still in the experimental phase, gene therapy holds transformative potential as a disease-modifying strategy capable of addressing the root causes of neuropsychiatric disorders. Further clinical validation and translational studies are essential to bridge the gap between laboratory success and real-world application.
Keywords : Gene therapy; Anxiety disorders; Epilepsy; HTR2A; Neuropeptide Y; AAV vectors
Cite This Article:
Please cite this article in press M. Ganga Raju et al., Gene Therapy Approaches
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