Transgenic mouse models of neurodegenerative disease: opportunities for therapeutic development

Curr Neurol Neurosci Rep. 2002 Sep;2(5):457-64. doi: 10.1007/s11910-002-0073-7.


Neurodegenerative diseases present an extraordinary challenge for medicine due to the grave nature of these illnesses, their prevalence, and their impact on individuals and caregivers. The most common of these age-associated chronic illnesses are Alzheimer's disease (AD) and Parkinson's disease (PD); other examples include the prion disorders, amyotrophic lateral sclerosis (ALS), and the trinucleotide (CAG) repeat diseases. All of these diseases are characterized by well-defined clinical syndromes with progressive courses that reflect the dysfunction and eventual loss of specific neuronal populations. Current therapies provide only symptomatic relief; none significantly alter the course of disease. We describe here how transgenic mice designed to model these diseases have substantially contributed to the identification and validation of many promising new therapies, and conversely how they have quickly and cost effectively eliminated several targets with unrealized expectations.

Publication types

  • Review

MeSH terms

  • Alzheimer Disease / genetics
  • Alzheimer Disease / metabolism
  • Alzheimer Disease / therapy
  • Amyloid beta-Peptides / metabolism
  • Amyotrophic Lateral Sclerosis / genetics
  • Amyotrophic Lateral Sclerosis / metabolism
  • Amyotrophic Lateral Sclerosis / therapy
  • Animals
  • Disease Models, Animal*
  • Huntington Disease / genetics
  • Huntington Disease / therapy
  • Mice
  • Mice, Transgenic
  • Motor Neuron Disease / genetics
  • Motor Neuron Disease / pathology
  • Motor Neuron Disease / therapy
  • Mutation
  • Nervous System / metabolism
  • Nervous System / pathology*
  • Neurodegenerative Diseases / genetics
  • Neurodegenerative Diseases / metabolism
  • Neurodegenerative Diseases / therapy*
  • Prion Diseases / genetics
  • Prion Diseases / metabolism
  • Prion Diseases / therapy
  • Spinocerebellar Degenerations / genetics
  • Spinocerebellar Degenerations / metabolism
  • Spinocerebellar Degenerations / therapy
  • Trinucleotide Repeats / genetics


  • Amyloid beta-Peptides