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Consensus Paper: Strengths and Weaknesses of Animal Models of Spinocerebellar Ataxias and Their Clinical Implications

J. Cendelin, M. Cvetanovic, M. Gandelman, H. Hirai, HT. Orr, SM. Pulst, M. Strupp, F. Tichanek, J. Tuma, M. Manto

. 2022 ; 21 (3) : 452-481. [pub] 20210810

Jazyk angličtina Země Spojené státy americké

Typ dokumentu časopisecké články

Perzistentní odkaz   https://www.medvik.cz/link/bmc22018112

Grantová podpora
R01 NS109077 NINDS NIH HHS - United States
18H02521 KAKENHI
R37NS033123 National Institutes of Health (USA)
U01NS103883 National Institutes of Health (NIH) / National Institute of Neurological Disorders and Stroke (NINDS)
JP20dm0207057 AMED
Brain/MINDS Japan Agency for Medical Research and Development
UO1NS103883 National Institutes of Health (USA)
R01 NS197387 National Institutes of Health (NIH) / National Institute of Neurological Disorders and Stroke (NINDS)
R21NSNS103009 National Institutes of Health (USA)
R01 NS097903 NINDS NIH HHS - United States
R37 NS033123 NINDS NIH HHS - United States
U01 NS103883 NINDS NIH HHS - United States
R01NS109077 National Institutes of Health (NIH) / National Institute of Neurological Disorders and Stroke (NINDS)
Q39 Univerzita Karlova v Praze
R37NS033123 National Institutes of Health (NIH) / National Institute of Neurological Disorders and Stroke (NINDS)
CZ.02.1.01/0.0/0.0/16_019/0000787 Ministerstvo Školství, Mládeže a Tělovýchovy
R01 NS107387 NINDS NIH HHS - United States
R01NS097903 National Institutes of Health (NIH) / National Institute of Neurological Disorders and Stroke (NINDS)
R21 NS103009 NINDS NIH HHS - United States
grants R21NS103009 National Institutes of Health (NIH) / National Institute of Neurological Disorders and Stroke (NINDS)

E-zdroje Online Plný text

NLK ProQuest Central od 2002-03-01 do Před 1 rokem
Medline Complete (EBSCOhost) od 2002-01-01 do Před 1 rokem
Nursing & Allied Health Database (ProQuest) od 2002-03-01 do Před 1 rokem
Health & Medicine (ProQuest) od 2002-03-01 do Před 1 rokem
Psychology Database (ProQuest) od 2002-03-01 do Před 1 rokem

Odkazy

PubMed 34378174
DOI 10.1007/s12311-021-01311-1
Knihovny.cz E-zdroje

Spinocerebellar ataxias (SCAs) represent a large group of hereditary degenerative diseases of the nervous system, in particular the cerebellum, and other systems that manifest with a variety of progressive motor, cognitive, and behavioral deficits with the leading symptom of cerebellar ataxia. SCAs often lead to severe impairments of the patient's functioning, quality of life, and life expectancy. For SCAs, there are no proven effective pharmacotherapies that improve the symptoms or substantially delay disease progress, i.e., disease-modifying therapies. To study SCA pathogenesis and potential therapies, animal models have been widely used and are an essential part of pre-clinical research. They mainly include mice, but also other vertebrates and invertebrates. Each animal model has its strengths and weaknesses arising from model animal species, type of genetic manipulation, and similarity to human diseases. The types of murine and non-murine models of SCAs, their contribution to the investigation of SCA pathogenesis, pathological phenotype, and therapeutic approaches including their advantages and disadvantages are reviewed in this paper. There is a consensus among the panel of experts that (1) animal models represent valuable tools to improve our understanding of SCAs and discover and assess novel therapies for this group of neurological disorders characterized by diverse mechanisms and differential degenerative progressions, (2) thorough phenotypic assessment of individual animal models is required for studies addressing therapeutic approaches, (3) comparative studies are needed to bring pre-clinical research closer to clinical trials, and (4) mouse models complement cellular and invertebrate models which remain limited in terms of clinical translation for complex neurological disorders such as SCAs.

Citace poskytuje Crossref.org

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