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Nihon Rinsho. 2005 Mar;63(3):394-400.

[Gene therapy for Alzheimer' s disease].

[Article in Japanese]

Author information

  • 1Laboratory for Proteolytic Neuroscience, RIKEN Brain Science Institute.


One of the keys to successful gene therapy is the selection of the appropriate therapeutic gene and its molecular vehicle. A recombinant adeno-associated virus (rAAV) vector offers the advantage of the ability to infect non-dividing cells, affording a non-pathogenic, long-term transgene expression without a substantial inflammatory response. In Alzheimer's disease (AD), accumulation of amyloid-beta peptide (Abeta) in the brain is a triggering event leading to the long-term pathological cascade. Therefore, it is necessary for therapy and prevention of AD to promote the degradation and clearance of Abeta . Neprilysin is a rate-limiting peptidase in Abeta degradation in the brain and a reduction in neprilysin activity will contribute to Abeta deposition and thus to AD development. As an experimental gene therapy for AD, neprilysin gene was introduced in the hippocampus of mice using rAAV vector. The neprilysin gene transfer increased the reduced neprilysin activity, abolished the increase of Abeta levels in the hippocampus of neprilysin-deficient mice, and also remarkably decelerated amyloid deposition in aged AD model mice. Thus, introduction of this Abeta--degrader gene into the brain would have therapeutic potential for AD.

[PubMed - indexed for MEDLINE]
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