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Mouse models of polyglutamine diseases in therapeutic approaches: review and data table. Part II
Pawel M Switonski1, Wojciech J Szlachcic, Agnieszka Gabka
1Institute of Bioorganic Chemistry, Polish Academy of Sciences, Noskowskiego 12/14, 61-704 Poznan, Poland.
Abstract:
Mouse models of human diseases are created both to understand the pathogenesis of the disorders and to find successful therapies for them. This work is the second part in a series of reviews of mouse models of polyglutamine (polyQ) hereditary disorders and focuses on in vivo experimental therapeutic approaches. Like part I of the polyQ mouse model review, this work is supplemented with a table that contains data from experimental studies of therapeutic approaches in polyQ mouse models. The aim of this review was to characterize the benefits and outcomes of various therapeutic strategies in mouse models. We examine whether the therapeutic strategies are specific to a single disease or are applicable to more than one polyQ disorder in mouse models. In addition, we discuss the suitability of mouse models in therapeutic approaches. Although the majority of therapeutic studies were performed in mouse models of Huntington disease, similar strategies were also used in other disease models.
Insights
This review examines therapeutic strategies in mouse models of polyglutamine (polyQ) diseases. It analyzes the effectiveness and disease specificity of various treatments, aiding in the development of therapies for these hereditary disorders.
Area of Science:
- Neuroscience
- Genetics
- Pharmacology
Background:
- Mouse models are crucial for understanding human disease pathogenesis and developing treatments.
- Polyglutamine (polyQ) disorders are a group of hereditary neurological diseases.
- This review focuses on experimental therapeutic approaches in polyQ mouse models.
Purpose of the Study:
- To characterize the benefits and outcomes of various therapeutic strategies in polyQ mouse models.
- To determine if therapeutic strategies are disease-specific or applicable across multiple polyQ disorders.
- To discuss the suitability of mouse models for evaluating therapeutic approaches.
Main Methods:
- Review of experimental studies on therapeutic approaches in polyQ mouse models.
- Data compilation in a supplementary table.
- Analysis of therapeutic strategy specificity and outcomes.
Main Results:
- Various therapeutic strategies have been tested in polyQ mouse models, with a majority focusing on Huntington disease models.
- Similar therapeutic strategies show applicability across different polyQ disorders.
- The suitability of mouse models for evaluating therapeutic approaches is discussed.
Conclusions:
- Mouse models provide valuable insights into the efficacy of therapeutic strategies for polyQ disorders.
- Understanding the benefits and outcomes of these strategies can guide future treatment development.
- Further research is needed to optimize therapeutic approaches and their translation to human patients.
