Standardization and statistical approaches to therapeutic trials in the R6/2 mouse

Brain Res Bull. 2003 Sep 30;61(5):469-79. doi: 10.1016/s0361-9230(03)00185-0.


The R6/2 mouse is the most widely used animal model of Huntington's disease (HD), a genetic disorder causing movement disorders, personality changes, dementia, and premature death, for which there is currently no effective therapy. Use of animal models to assess novel therapeutic approaches to HD is currently a major focus of research. Progress in this field will depend upon careful standardization of experimental protocols, and a sophisticated statistical approach. Here we investigate the sources of phenotypic variability in R6/2, and make recommendations for the future use of such models in therapeutic trials.

Publication types

  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, Non-P.H.S.

MeSH terms

  • Animals
  • Body Weight / genetics
  • Disease Models, Animal*
  • Drug Evaluation, Preclinical / methods
  • Drug Evaluation, Preclinical / standards*
  • Drug Evaluation, Preclinical / statistics & numerical data
  • Female
  • Genetic Variation / genetics*
  • Huntington Disease / drug therapy*
  • Huntington Disease / genetics
  • Male
  • Mice
  • Mice, Mutant Strains
  • Models, Statistical
  • Motor Activity / genetics
  • Peptides / genetics
  • Phenotype
  • Polymorphism, Genetic / genetics
  • Research Design / standards
  • Research Design / statistics & numerical data
  • Siblings
  • Species Specificity*
  • Trinucleotide Repeats / genetics


  • Peptides
  • polyglutamine