Gene Delivery and Gene Therapy for Alzheimer’s Disease

  • Maya Woodbury
  • Tomomi Kiyota
  • Tsuneya IkezuEmail author
Part of the Neuromethods book series (NM, volume 98)


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 intensified. 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.

Key words

Adeno-associated virus Alzheimer’s disease Gene therapy 


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Copyright information

© Springer Science+Business Media New York 2015

Authors and Affiliations

  1. 1.Department of Pharmacology and Experimental Therapeutics and Graduate Program in NeuroscienceBoston University School of MedicineBostonUSA
  2. 2.Department of Pharmacology and Experimental NeuroscienceUniversity of Nebraska Medical CenterOmahaUSA
  3. 3.Department of NeurologyBoston University School of MedicineBostonUSA
  4. 4.Laboratory of Molecular NeuroTherapeutics, Department of Pharmacology and Experimental Therapeutics and NeurologyBoston University School of MedicineBostonUSA

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