K004 Advanced therapies working group: task force for preclinical models of Huntington’s disease

<h3>Background</h3> A wide variety of rodent models exist for Huntington’s disease, including toxin-induced models, transgenic rodents and knock-in models. These models have been important in advancing our understanding of the biological changes associated with the disease and they present with many relevant behavioural features of the human condition. Despite this, there is a recognition that the models vary widely in their utility to address specific scientific questions. Moreover, thus far, many therapeutics that have shown efficacy in rodent models have failed to translate to the clinical, raising questions about their face validity and predicative validity. <h3>Aim</h3> To establish a task force that will consider the role of preclinical rodent models in HD research. <h3>Methods/Results</h3> We have started to bring together scientists with experience of using mouse and rat models of HD in their research, and we welcome engagement from new scientists. We will hold meetings to discuss key questions in the field, and we have started to prepare a manuscript that will consider in detail how well biological and behavioural features of disease are represented in our current models. We also aim to consider the future of preclinical research and how we must progress the field in order to generate effective therapeutics in preclinical models of HD. <h3>Conclusion</h3> Despite significant advances in developing relevant preclinical models of HD, we are at a stage in which we need to work together as a field to better understand their limitations and to develop strategies to optimise preclinical research for HD.

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Published
2024-09-01
DOI
https://doi.org/10.1136/jnnp-2024-ehdn.353
Primary Topic
Genetic Neurodegenerative Diseases
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article
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article

K004 Advanced therapies working group: task force for preclinical models of Huntington’s disease

Mariah J. Lelos, Sandrine Humbert
Genetic Neurodegenerative Diseases
article

K004 Advanced therapies working group: task force for preclinical models of Huntington’s disease

Mariah J. Lelos, Sandrine Humbert
article en

Abstract

<h3>Background</h3> A wide variety of rodent models exist for Huntington’s disease, including toxin-induced models, transgenic rodents and knock-in models. These models have been important in advancing our understanding of the biological changes associated with the disease and they present with many relevant behavioural features of the human condition. Despite this, there is a recognition that the models vary widely in their utility to address specific scientific questions. Moreover, thus far, many therapeutics that have shown efficacy in rodent models have failed to translate to the clinical, raising questions about their face validity and predicative validity. <h3>Aim</h3> To establish a task force that will consider the role of preclinical rodent models in HD research. <h3>Methods/Results</h3> We have started to bring together scientists with experience of using mouse and rat models of HD in their research, and we welcome engagement from new scientists. We will hold meetings to discuss key questions in the field, and we have started to prepare a manuscript that will consider in detail how well biological and behavioural features of disease are represented in our current models. We also aim to consider the future of preclinical research and how we must progress the field in order to generate effective therapeutics in preclinical models of HD. <h3>Conclusion</h3> Despite significant advances in developing relevant preclinical models of HD, we are at a stage in which we need to work together as a field to better understand their limitations and to develop strategies to optimise preclinical research for HD.

Centre National de la Recherche Scientifique (FR), Inserm (FR), Sorbonne Université (FR), Institut du Cerveau (FR), Cardiff University (GB)
Openalex Percentile: Top 85%
Genetic Neurodegenerative Diseases
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K004 Advanced therapies working group: task force for preclinical models of Huntington’s disease — Mariah J. Lelos, Sandrine Humbert · (2024) | TGRS Research Map | TGRS