Modelling C9orf72-Related Amyotrophic Lateral Sclerosis in Zebrafish

Fortier, Gabrielle; Butti, Zoé; Patten, Scott B.

doi:10.3390/biomedicines8100440

Cited by 10 publications

(10 citation statements)

References 102 publications

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“…Knockdown of the C9ORF72 zebrafish ortholog induces axonopathy of motor neurons and locomotor deficits that, of note, are rescued by overexpressing human C9ORF72 mRNA transcripts [ 194 , 195 ]. Conversely, G 4 C 2 HRE results in RNA foci and dipeptide repeat formation leading to a significant increase of apoptotic cells, toxicity and motor axon abnormalities [ 196 , 197 , 198 ].…”

Section: Modeling Als In Different Systemsmentioning

confidence: 99%

Where and Why Modeling Amyotrophic Lateral Sclerosis

Liguori

Amadio

Volonté

2021

IJMS

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Over the years, researchers have leveraged a host of different in vivo models in order to dissect amyotrophic lateral sclerosis (ALS), a neurodegenerative/neuroinflammatory disease that is heterogeneous in its clinical presentation and is multigenic, multifactorial and non-cell autonomous. These models include both vertebrates and invertebrates such as yeast, worms, flies, zebrafish, mice, rats, guinea pigs, dogs and, more recently, non-human primates. Despite their obvious differences and peculiarities, only the concurrent and comparative analysis of these various systems will allow the untangling of the causes and mechanisms of ALS for finally obtaining new efficacious therapeutics. However, harnessing these powerful organisms poses numerous challenges. In this context, we present here an updated and comprehensive review of how eukaryotic unicellular and multicellular organisms that reproduce a few of the main clinical features of the disease have helped in ALS research to dissect the pathological pathways of the disease insurgence and progression. We describe common features as well as discrepancies among these models, highlighting new insights and emerging roles for experimental organisms in ALS.

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Section: Modeling Als In Different Systemsmentioning

confidence: 99%

Where and Why Modeling Amyotrophic Lateral Sclerosis

Liguori

Amadio

Volonté

2021

IJMS

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“…Zebrafish (Danio rerio) is often used to study embryonic development because of the transparency of their embryos and their vertebrate body plan [275] and it is increasingly being used also in ALS [216,[276][277][278]. Most human genes have a zebrafish homologue, typically with approximately 70% homology in protein sequence [275].…”

Section: Danio Rerio (Zebrafish) Modelsmentioning

confidence: 99%

Nearly 30 Years of Animal Models to Study Amyotrophic Lateral Sclerosis: A Historical Overview and Future Perspectives

Bonifacino

Zerbo

Balbi

et al. 2021

IJMS

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Amyotrophic lateral sclerosis (ALS) is a fatal, multigenic, multifactorial, and non-cell autonomous neurodegenerative disease characterized by upper and lower motor neuron loss. Several genetic mutations lead to ALS development and many emerging gene mutations have been discovered in recent years. Over the decades since 1990, several animal models have been generated to study ALS pathology including both vertebrates and invertebrates such as yeast, worms, flies, zebrafish, mice, rats, guinea pigs, dogs, and non-human primates. Although these models show different peculiarities, they are all useful and complementary to dissect the pathological mechanisms at the basis of motor neuron degeneration and ALS progression, thus contributing to the development of new promising therapeutics. In this review, we describe the up to date and available ALS genetic animal models, classified by the different genetic mutations and divided per species, pointing out their features in modeling, the onset and progression of the pathology, as well as their specific pathological hallmarks. Moreover, we highlight similarities, differences, advantages, and limitations, aimed at helping the researcher to select the most appropriate experimental animal model, when designing a preclinical ALS study.

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“…It has been reported that zebrafish harboring human ALS mutant genes can serve as a model animal to study MN degeneration [ 93 , 94 ]. For instance, overexpression of mutant human SOD 1 ( Cu-Zn superoxide dismutase 1 ) in zebrafish led to short motor axons with premature branching accompanied by deficient locomotion [ 95 ].…”

Section: Rare Disease and Screening For Potential Drugs Or Proteinsmentioning

confidence: 99%

Zebrafish, an In Vivo Platform to Screen Drugs and Proteins for Biomedical Use

Lin

Tsai

2021

Pharmaceuticals

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The nearly simultaneous convergence of human genetics and advanced molecular technologies has led to an improved understanding of human diseases. At the same time, the demand for drug screening and gene function identification has also increased, albeit time- and labor-intensive. However, bridging the gap between in vitro evidence from cell lines and in vivo evidence, the lower vertebrate zebrafish possesses many advantages over higher vertebrates, such as low maintenance, high fecundity, light-induced spawning, transparent embryos, short generation interval, rapid embryonic development, fully sequenced genome, and some phenotypes similar to human diseases. Such merits have popularized the zebrafish as a model system for biomedical and pharmaceutical studies, including drug screening. Here, we reviewed the various ways in which zebrafish serve as an in vivo platform to perform drug and protein screening in the fields of rare human diseases, social behavior and cancer studies. Since zebrafish mutations faithfully phenocopy many human disorders, many compounds identified from zebrafish screening systems have advanced to early clinical trials, such as those for Adenoid cystic carcinoma, Dravet syndrome and Diamond–Blackfan anemia. We also reviewed and described how zebrafish are used to carry out environmental pollutant detection and assessment of nanoparticle biosafety and QT prolongation.

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Modelling C9orf72-Related Amyotrophic Lateral Sclerosis in Zebrafish

Cited by 10 publications

References 102 publications

Where and Why Modeling Amyotrophic Lateral Sclerosis

Where and Why Modeling Amyotrophic Lateral Sclerosis

Nearly 30 Years of Animal Models to Study Amyotrophic Lateral Sclerosis: A Historical Overview and Future Perspectives

Zebrafish, an In Vivo Platform to Screen Drugs and Proteins for Biomedical Use

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