Potential Gene Therapy Towards Treating Neurodegenerative Disea ses Employing Polymeric Nanosystems

Curr Gene Ther. 2017;17(2):170-183. doi: 10.2174/1566523217666170510153845.


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.

Keywords: Alzheimer's diseases; Amyotrophic lateral sclerosis; Blood brain barrier; Gene therapy; Huntington's disease; Parkinson's disease; Polymeric nanoparticles.

Publication types

  • Review

MeSH terms

  • Animals
  • Drug Carriers / chemistry
  • Drug Delivery Systems / methods
  • Gene Transfer Techniques
  • Genetic Therapy / methods*
  • Humans
  • Nanoparticles / administration & dosage*
  • Nanoparticles / chemistry
  • Nanotechnology / methods
  • Neurodegenerative Diseases / genetics
  • Neurodegenerative Diseases / therapy*
  • Polymers / administration & dosage*
  • Polymers / chemistry


  • Drug Carriers
  • Polymers