Gene delivery and gene therapy for Alzheimer’s disease

Maya Woodbury, Tomomi Kiyota, Tsuneya Ikezu

Research output: Contribution to journalArticle

Abstract

Adeno-associated virus (AAV) is the most widely used vector for gene therapy clinical trials focused on neurodegeneration. Not only can it safely and efficiently confer long-term expression in a variety of cell types, but innovations including hybrid serotypes, rationally designed capsids, split vectors, specific promoter/enhancer additions, and convection-enhanced delivery have greatly progressed the field of AAV research for neurodegenerative disease. Indeed, these developments, which enable increased specificity, efficiency, and spread of gene transfer as well as ease of administration to the CNS, have pushed AAV forward into clinical trials for Alzheimer’s disease. Preclinical testing of AAV gene transfer efficacy in animal models of disease has thus intensifi ed. In this chapter, we provide detailed methods for construction of AAV expression vectors, their application in preclinical studies using established animal models of Alzheimer’s disease, and evaluation methods to assess Alzheimer’s disease-like behavior and brain pathology.

Original languageEnglish (US)
Pages (from-to)85-120
Number of pages36
JournalNeuromethods
Volume98
DOIs
StatePublished - Jan 1 2015

Fingerprint

Gene therapy
Dependovirus
Viruses
Genetic Therapy
Alzheimer Disease
Genes
Gene transfer
Animals
Clinical Trials
Neurodegenerative diseases
Animal Disease Models
Convection
Capsid
Pathology
Neurodegenerative Diseases
Brain
Animal Models
Innovation
Testing
Research

Keywords

  • Adeno-associated virus
  • Alzheimer’s disease
  • Gene therapy

ASJC Scopus subject areas

  • Neuroscience(all)
  • Biochemistry, Genetics and Molecular Biology(all)
  • Pharmacology, Toxicology and Pharmaceutics(all)
  • Psychiatry and Mental health

Cite this

Gene delivery and gene therapy for Alzheimer’s disease. / Woodbury, Maya; Kiyota, Tomomi; Ikezu, Tsuneya.

In: Neuromethods, Vol. 98, 01.01.2015, p. 85-120.

Research output: Contribution to journalArticle

Woodbury, Maya ; Kiyota, Tomomi ; Ikezu, Tsuneya. / Gene delivery and gene therapy for Alzheimer’s disease. In: Neuromethods. 2015 ; Vol. 98. pp. 85-120.
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