Intracranial adeno-associated virus-mediated delivery of anti-pan amyloid beta, amyloid beta40, and amyloid beta42 single-chain variable fragments attenuates plaque pathology in amyloid precursor protein mice

J Neurosci. 2006 Nov 15;26(46):11923-8. doi: 10.1523/JNEUROSCI.2795-06.2006.

Abstract

Accumulation of amyloid beta protein (Abeta) aggregates is hypothesized to trigger a pathological cascade that causes Alzheimer's disease (AD). Active or passive immunizations targeting Abeta are therefore of great interest as potential therapeutic strategies. We have evaluated the use of recombinant anti-Abeta single-chain variable fragments (scFvs) as a potentially safer form of anti-Abeta immunotherapy. We have generated and characterized three anti-Abeta scFvs that recognize Abeta 1-16, Abeta x-40, or Abeta x-42. To achieve widespread brain delivery, constructs expressing these anti-Abeta scFvs were packaged into adeno-associated virus (AAV) vectors and injected into the ventricles of postnatal day 0 (P0) amyloid precursor protein CRND8-transgenic mice. Intracranial delivery of AAV to neonatal mice resulted in widespread neuronal delivery. In situ expression of each of the anti-Abeta scFvs after intracerebroventricular AAV serotype 1 delivery to P0 pups decreased Abeta deposition by 25-50%. These data suggest that intracranial anti-Abeta scFv expression is an effective strategy to attenuate amyloid deposition. As opposed to transgenic approaches, these studies also establish a "somatic brain transgenic" paradigm to rapidly and cost-effectively evaluate potential modifiers of AD-like pathology in AD mouse models.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Alzheimer Disease / immunology
  • Alzheimer Disease / physiopathology
  • Alzheimer Disease / therapy*
  • Amyloid beta-Peptides / antagonists & inhibitors*
  • Amyloid beta-Peptides / immunology
  • Amyloid beta-Peptides / toxicity
  • Amyloid beta-Protein Precursor / genetics
  • Amyloid beta-Protein Precursor / metabolism
  • Animals
  • Animals, Newborn
  • Antibodies / chemistry
  • Antibodies / immunology*
  • Brain / drug effects
  • Brain / immunology
  • Brain / physiopathology
  • Dependovirus / immunology*
  • Disease Models, Animal
  • Female
  • Genetic Therapy / methods*
  • Immunoglobulin Variable Region / chemistry
  • Immunoglobulin Variable Region / genetics
  • Immunoglobulin Variable Region / immunology
  • Injections, Intraventricular
  • Male
  • Mice
  • Mice, Mutant Strains
  • Mice, Transgenic
  • Neurons / drug effects
  • Neurons / immunology
  • Neurons / pathology
  • Peptide Fragments / antagonists & inhibitors
  • Peptide Fragments / chemistry
  • Peptide Fragments / genetics
  • Peptide Fragments / immunology
  • Peptide Fragments / toxicity
  • Plaque, Amyloid / immunology*
  • Plaque, Amyloid / pathology
  • Treatment Outcome

Substances

  • Amyloid beta-Peptides
  • Amyloid beta-Protein Precursor
  • Antibodies
  • Immunoglobulin Variable Region
  • Peptide Fragments
  • amyloid beta-protein (1-40)
  • amyloid beta-protein (1-42)