The Role of Animal Models in Huntington's Disease Clinical Trials: Decoding Genetic, Non-Genetic, and Molecular Pathways
Abstract
BACKGROUND
Huntington's disease (HD) is a neurodegenerative disorder due to a CAG trinucleotide repeat expansion in the HD gene. Animal models have been instrumental in revealing the genetic and molecular bases of HD. While animal models cannot exactly model the human disease because of anatomical and lifespan differences, they are essential in revealing HD pathology and possible treatments.
OBJECTIVE
This review aimed to highlight the significance of animal models, particularly rodents, in deepening our knowledge of Huntington's disease. It underlines how non-genetic and genetic models have aided research and therapy innovation as well as their limitations.
METHODS
This review addresses the use of different models of animals, including genetic models, such as transgenic mice and non-genetic models, for example, invertebrates and non-human primates. It addresses the creation of these models through methods, such as gene transfer techniques and transgenic manipulation, to simulate the genetic defects that occur in humans. The applicability of model choice based on validity criteria, including symptom manifestation and treatment effectiveness, is also discussed.
RESULTS
This study underscores the effectiveness of the R6/2 mouse model, characterized by accelerated symptom onset and HD pathology. Progress in genetic engineering has also boosted the construction of murine and rat models that reproduce the hereditary aspects of HD, providing significant platforms for experimental…
Read the paper
Related studies
- Improving sleep quality leads to better mental health: A meta-analysis of randomised controlled trials
- Tocilizumab in patients with severe COVID-19: a retrospective cohort study
- A new aging measure captures morbidity and mortality risk across diverse subpopulations from NHANES IV: A cohort study
- Fruit, vegetable, and legume intake, and cardiovascular disease and deaths in 18 countries (PURE): a prospective cohort study
- Are noise and air pollution related to the incidence of dementia? A cohort study in London, England
- Long COVID symptoms and duration in SARS-CoV-2 positive children — a nationwide cohort study
Go deeper in Evidence Hub
Evidence Hub adds a study data sheet (design, sample size, evidence level, key numbers), an AI summary and a chat grounded in the paper's text.