Delivery Methods for Treatment of Genetic Disorders

Kunal Gupta, Nathan R. Selden

Research output: Chapter in Book/Report/Conference proceedingChapter


The effective delivery of gene therapy can be reduced to three essential components: (1) specific targeting of an anatomical region or cell population, (2) transfer of genetic material to the host, and (3) efficacious expression that alters the disease phenotype. The predominant delivery methods are viral-mediated and nonviral-mediated therapies. Each method has benefits and caveats that affect the targeting of specific regions, stable or unstable transfer of genetic material, and effect on the targeted disorder. In this chapter we will discuss viral- and nonviral-mediated therapies, challenges in applying these therapies in animal models and human patients, and ways to circumvent these delivery roadblocks. We will also discuss specific adaptations of these methods in attempting delivery to the central nervous system.

Original languageEnglish (US)
Title of host publicationNervous System Drug Delivery
Subtitle of host publicationPrinciples and Practice
Number of pages15
ISBN (Electronic)9780128139981
ISBN (Print)9780128139974
StatePublished - Jun 25 2019


  • CRISPR/Cas9
  • Central nervous system
  • Gene therapy
  • Stem cells
  • Stereotactic surgery
  • Virus

ASJC Scopus subject areas

  • Medicine(all)


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