Title:Nanoparticle Mediated Gene Therapy: A Trailblazer Armament to
Fight CNS Disorders
Volume: 30
Issue: 3
Author(s): Annu, Saleha Rehman, Bushra Nabi, Ali Sartaj, Shadab Md, PK Sahoo, Sanjula Baboota and Javed Ali*
Affiliation:
- Department of Pharmaceutics, School of Pharmaceutical Education and Research, Jamia Hamdard, New
Delhi-110062, India
Keywords:
Gene therapy, nanoparticles, Parkinson’s disease, Alzheimer’s disease, stroke, brain tumors.
Abstract: Central nervous system (CNS) disorders account for boundless socioeconomic
burdens with devastating effects among the population, especially the elderly. The major
symptoms of these disorders are neurodegeneration, neuroinflammation, and cognitive
dysfunction caused by inherited genetic mutations or by genetic and epigenetic changes
due to injury, environmental factors, and disease-related events. Currently available clinical
treatments for CNS diseases, i.e., Alzheimer’s disease, Parkinson’s disease, stroke,
and brain tumor, have significant side effects and are largely unable to halt the clinical
progression. So gene therapy displays a new paradigm in the treatment of these disorders
with some modalities, varying from the suppression of endogenous genes to the expression
of exogenous genes. Both viral and non-viral vectors are commonly used for gene
therapy. Viral vectors are quite effective but associated with severe side effects, like immunogenicity
and carcinogenicity, and poor target cell specificity. Thus, non-viral vectors,
mainly nanotherapeutics like nanoparticles (NPs), turn out to be a realistic approach
in gene therapy, achieving higher efficacy. NPs demonstrate a new avenue in pharmacotherapy
for the delivery of drugs or genes to their selective cells or tissue, thus providing
concentrated and constant drug delivery to targeted tissues, minimizing systemic
toxicity and side effects. The current review will emphasize the role of NPs in mediating
gene therapy for CNS disorders treatment. Moreover, the challenges and perspectives of
NPs in gene therapy will be summarized.