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Animal Models for Huntington's Disease

Animal Models for Huntington’s Disease

The lack of suitable animal models for therapeutic drug studies is one of the reasons why it is so difficult to discover successful treatments for Huntington's disease (HD). Our company is committed to providing our clients with a range of disease modeling services and in vivo animal studies to provide reliable preclinical research data. Our services are intended to help you validate your targets or advance your lead candidates. No matter what stage of your HD program, we offer predictable translational HD research to enhance your program's efficacy.


HD is a progressive neurodegenerative disease that affects approximately 1 in 10,000 individuals worldwide. It is caused by the amplification of a polyglutamine repeat in the disease protein huntingtin (HTT), leading to the accumulation of toxic protein aggregates in the brain and ultimately resulting in the loss of neurons and the development of motor, cognitive, and psychiatric symptoms. Despite extensive research, there is no cure for HD and the available treatments only provide symptomatic relief.

Animal models are important tools for uncovering the pathogenesis and therapeutic targets of neurodegenerative diseases. In the case of HD, animal models have been particularly important due to the limitations of studying the disease in humans. Currently, animal models of HD have been established in a variety of species, such as fruit flies, worms, rodents, pigs, sheep, and monkeys. These models provide significant insights into the pathogenesis of this disease and the effectiveness of candidate therapies.

Fig. 1 Apoptotic cells in the brains of transgenic HD pigs.

Fig. 1 Apoptotic cells in the brains of transgenic HD pigs. (Li X J, et al., 2015)

Our Methods

We help our clients generate animal models of HD by multiple genetic approaches and we select the appropriate method according to their specific purpose and question of interest. The key distinguishing factors between these approaches include the following:

  • Use of full-length or only fragments of mutant HTT
  • Different lengths of CAG repeat sequences incorporated into the genetic construct
  • Use of a coding region containing only CAG repeats or a coding region containing repeats interrupted by one or more CAA codons
  • Expression of HD mutations from a transgene or knock-in of the mutation into the endogenous Htt locus
  • Use of human HTT or endogenous genes from animals
  • Use of cDNA or genomic DNA containing all introns and regulatory sequences
  • Use of HTT promoter or another promoter to drive expression of mutant proteins

Disease Modeling Services

We are committed to providing our clients with a variety of HD animal models and assessing the translational potential of animal models based on their construct, face, and predictive validity. We select the right animal model for our clients based on the strengths of the various model systems and our knowledge of the phenotypes and endpoints characterized in these models. In addition to the most common animal models such as rodents, we also provide large animal models for our clients to study the pathogenesis of HD and to develop new therapeutic strategies. Our animal models include, but are not limited to:

  • Transgenic C. elegans models
    • Truncated HTT N-terminal fragment models: HtnQ95, HtnQ150, Htt57Q88, Htt57Q128
  • D. melanogaster models
    • Truncated HTT N-terminal fragment models: HttQ75, HttQ120, Httex1pQ93, Htt128Q, N-termHttQ128
    • Full-length HTT models: Htt128QFL
  • Rodent models
    • Truncated N-terminal fragment models: R6/1 mice, R6/2 mice, N171-Q82 mice, HD94 mice, Shortstop mice, N118- 82Q mice, N586- 82Q mice, Ubi-G-HTT84Q mice, HD51 rats
    • Knock-in models: CAG140 mice, HdhQ92 mice, HdhQ111 mice, HdhQ150 mice, HdhQ200 mice, Hdh4/Q72 mice, Hdh4/Q80 mice, zQ175 mice
    • Transgenic models: HD48 mice, HD89 mice, YAC128 mice, YAC48 mice, YAC72 mice, BACHD mice, Hu97/18 mice, iFL148Q mice, BACHD rats
  • Large animal models
    • Sheep models: OVT73 transgenic mHTT sheep, knock-in mHtt sheep
    • Minipig models: transgenic mHTT minipig, KI-HD-85Q knock-in mHtt minipig, KI-HD-150Q knock-in mHtt minipig
    • Nonhuman primate (NHP) models: AAV-mHTT somatic models, knock-in mHTT NHP

Our company is a leader in the field of HD research and offers a range of disease modeling and preclinical research services to support the development of new treatments. Our expertise, commitment to ethical and humane animal research, and state-of-the-art facilities make us the ideal partner for any HD research project. If you are interested in our disease modeling services, please contact us for more information.


  • Li, X. J.; Li, S. Large animal models of Huntington's disease. Behavioral Neurobiology of Huntington's Disease and Parkinson's Disease, 2015: 149-160.

All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.

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