Background: Recent integrated approaches involving nanotechnology and gene therapy
have accelerated development of efficient drug delivery to the central nervous system (CNS). Neurodegenerative
disorders are closely associated with genetic inheritance and mutation.
Materials: Nanotechnology has allowed effective engineering of various such polymeric structures.
Moreover, availability of a wide array of polymeric materials has enabled fabrication of biocompatible
and biodegradable delivery vehicles. Our manuscript focuses on the ideal features and properties of
polymeric nanoparticles that have enabled successful gene therapy for neurodegenerative disorders, as
well as the challenges that are posing difficulties in their practical application. We have highlighted
these aspects through examples of polymeric nanoparticles that have exhibited therapeutic promise in
the treatment of neurological disorders and mutations.
Methods: Complete cure of these diseases is a challenging task and gene therapy appears as a realistic
approach for their treatment. Gene therapy allows effective replacement or suppression of faulty
genes, thereby increasing chances for neuron survival and repair. However, successful delivery of naked
genetic material to CNS faces severe obstacles due to possible degradation and restricted transportation
of these biological entities across the blood brain barrier (BBB). Structurally, the BBB is composed
of several tight junctions, making the membrane highly selective towards the entry of molecules.
Conclusion: In order to target BBB for treating neurodegenerative diseases, it is essential to develop a
tailor-made system that may not only cross this barrier, but also effectively modulate the expression of
disease-causing genes. Stabilization of therapeutic genes and their effective, targeted delivery may be
possible using polymeric nanoparticles as carriers.