Adeno-associated virus-mediated gene therapy in central nervous system genetic disorders

Widler Casy, Qinglan Ling, Frances C. Shaffo, Sarah E. Sinnett, Steven J. Gray

Research output: Chapter in Book/Report/Conference proceedingChapter


Gene therapy has become a promising strategy to treat the genetic disorders of central nervous system (CNS). In the last five decades the development and characterization of recombinant adeno-associated virus (rAAV) as a safe and effective vector for gene delivery has revolutionized this field. Development of rAAV with better efficacy and selectivity for the CNS has resulted in increasingly successful preclinical and clinical applications. This chapter provides an overview of AAV-mediated CNS gene therapy, including discovery and engineering of novel AAV capsids, advances in preclinical models of genetic disorders, and clinical translation of AAV technology.

Original languageEnglish (US)
Title of host publicationRosenberg’s Molecular and Genetic Basis of Neurological and Psychiatric Disease
Subtitle of host publicationVolume 1
Number of pages16
ISBN (Electronic)9780128139554
StatePublished - Jan 1 2020


  • AAV
  • Capsid engineering
  • Central nervous system
  • Gene therapy
  • Rare genetic disorders

ASJC Scopus subject areas

  • Medicine(all)


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