Gene therapy for neurological disorders: challenges and recent advancements

Stefanie A. Pena, Rahul Iyengar, Rebecca S. Eshraghi, Nicole Bencie, Jeenu Mittal, Abdulrahman Aljohani, Rahul Mittal, Adrien A. Eshraghi

Research output: Contribution to journalReview articlepeer-review

4 Scopus citations


Major advancements in targeted gene therapy have opened up avenues for the treatment of major neurological disorders through a range of versatile modalities varying from expression of exogenous to suppression of endogenous genes. Recent technological innovations for improved gene sequence delivery have focussed on highly specific viral vector designs, plasmid transfection, nanoparticles, polymer-mediated gene delivery, engineered microRNA and in vivo clustered regulatory interspaced short palindromic repeats (CRISPR)-based therapeutics. These advanced techniques have profound applications in treating highly prevalent neurological diseases and neurodevelopmental disorders including Parkinson’s disease, Alzheimer’s disease and autism spectrum disorder, as well as rarer diseases such as amyotrophic lateral sclerosis (ALS), spinal muscular atrophy, lysosomal storage diseases, X-linked adrenoleukodystrophy and oncological diseases. In this article, we present an overview of the latest advances in targeted gene delivery and discuss the challenges and future direction of gene therapy in the treatment of neurological disorders.

Original languageEnglish (US)
Pages (from-to)111-128
Number of pages18
JournalJournal of Drug Targeting
Issue number2
StatePublished - Feb 7 2020


  • Autism Spectrum disorder
  • CRISPER/Cas9 system
  • Gene therapy
  • gene delivery
  • neurological diseases
  • non-viral vectors
  • viral vectors

ASJC Scopus subject areas

  • Pharmaceutical Science


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