The neuropsychiatric illnesses have been enigmatic, with no effective treatment to date.
The complexity and heterogeneity of psychiatric disorders are daunting for the development of novel
treatment modalities. The conventional treatment approaches are less effective and are associated with
several side effects, thus creating the need for the development of more innovative strategies. Since
psychiatric disorders are known to exhibit genetic linkage, gene therapy has created an interest among
the researchers worldwide. The delivery of nucleic acids is a complex process requiring the transport
of genetic material across various intracellular and extracellular barriers to reach the target cells
eliciting the transfection process. Therefore, the identification or development of the delivery system
for nucleic acid delivery still remains the challenge. Viral vectors are quite effective but are associated
with toxicity and side effects. With the rapid advancement in the field of nanotechnology, nanosized
materials were identified to be the perfect candidate for nonviral vectors in gene delivery. The biggest
advantage of nanoparticles is that their surface can be engineered in many possible ways to deliver the
drugs directly to the target site. Although gene therapy has already been established as an innovative
treatment modality for several neurological diseases, its use in psychiatry still warrants more
investigations for its translation into clinical use. The present manuscript discusses the prospects of
gene therapy in psychiatric disorders, their benefits, and pitfalls. The review embarks upon the
importance of nanoparticle-based gene therapy for effective management of psychiatric disorders.
Keywords: Psychiatric disorders, BDNF, CRISPR, RNA, lipid nanoparticles, mesenchymal cells.
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