Gene therapy has a strong potential in neuroscience by suppressing or replacing abnormalities
in genetic materials. The employment of nano-gene carrier for neurological disorders is comparatively
young and restricted since the aim to effectively deliver therapeutic agents into the central
nervous system (CNS) commonly has confronted difficulties of several natural occurring barriers in
the body and unfavorable characteristics of pharmaceutical agents. Two major anatomical and biochemical
barriers are blood-brain barrier (BBB) and blood-cerebrospinal fluid barrier (BCSFB),
which significantly prevent permeation of most drugs and genes to brain parenchyma. In this concern,
nanotechnology emerges as an innovative method for transporting therapeutics to the CNS. Diverse
nano-systems have been closely investigated, some of which have demonstrated initial success for in
vivo studies. The perspectives of nanotechnology for gene therapy would be a promising field to be
further explored in the near future.
Keywords: Nanoparticles, gene therapy, blood-brain barrier, gene therapy, neurological disorders.
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