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Preclinical models: needed in translation? A Pro/Con debate.

Thomas Philips¹, Jeffrey D Rothstein, Mahmoud A Pouladi

¹Johns Hopkins University, Brain Science Institute, Baltimore, Maryland, USA.

Movement Disorders : Official Journal of the Movement Disorder Society

|September 13, 2014

Summary

Genetic animal models offer insights into neurodegenerative diseases like Huntington's disease (HD). However, despite their value, these models have limitations in developing effective HD treatments.

Keywords:

Animal models HTT Huntington's transgenic translation

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Area of Science:

Neuroscience
Genetics
Pharmacology

Background:

Neurodegenerative disorders, including Huntington's disease (HD), have numerous identified genetic mutations and risk factors.
Over 20 transgenic rodent models for HD have been created to study disease mechanisms.
These models have provided valuable insights but have not yet led to effective HD therapies.

Purpose of the Study:

To critically evaluate the utility of animal models in translational research for Huntington's disease.
To highlight the strengths and limitations of current genetic animal models for HD.

Main Methods:

Review of existing literature on genetic animal models for neurodegenerative diseases, with a focus on Huntington's disease.
Analysis of the contribution of these models to understanding disease mechanisms.
Assessment of the translational value of animal models in the context of therapeutic development for HD.

Main Results:

Genetic animal models have significantly advanced the understanding of neurodegenerative disease mechanisms.
Despite extensive research using these models, no effective treatments for Huntington's disease have been developed.
The predictive value of animal models for human therapeutic outcomes remains a significant challenge.

Conclusions:

Animal models are crucial for dissecting disease pathogenesis and identifying potential therapeutic targets.
Translational gaps persist between findings in animal models and clinical efficacy in humans for HD.
Further refinement of animal models and research strategies is needed to improve the translation of basic science discoveries into effective Huntington's disease treatments.