Contributions of Animal Models to the Mechanisms and Therapies of Transthyretin Amyloidosis
Transthyretin amyloidosis (ATTR amyloidosis) is a fatal systemic disease caused by amyloid deposits of misfolded transthyretin, leading to familial amyloid polyneuropathy and/or cardiomyopathy, or a rare oculoleptomeningeal amyloidosis. A good model system that mimic the disease phenotype is crucial...
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| Format: | Article |
| Language: | English |
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Frontiers Media S.A.
2019-04-01
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| Series: | Frontiers in Physiology |
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| Online Access: | https://www.frontiersin.org/article/10.3389/fphys.2019.00338/full |
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| author | Ridwan Babatunde Ibrahim Ridwan Babatunde Ibrahim Yo-Tsen Liu Yo-Tsen Liu Yo-Tsen Liu Yo-Tsen Liu Ssu-Yu Yeh Jin-Wu Tsai Jin-Wu Tsai |
| author_facet | Ridwan Babatunde Ibrahim Ridwan Babatunde Ibrahim Yo-Tsen Liu Yo-Tsen Liu Yo-Tsen Liu Yo-Tsen Liu Ssu-Yu Yeh Jin-Wu Tsai Jin-Wu Tsai |
| author_sort | Ridwan Babatunde Ibrahim |
| collection | DOAJ |
| description | Transthyretin amyloidosis (ATTR amyloidosis) is a fatal systemic disease caused by amyloid deposits of misfolded transthyretin, leading to familial amyloid polyneuropathy and/or cardiomyopathy, or a rare oculoleptomeningeal amyloidosis. A good model system that mimic the disease phenotype is crucial for the development of drugs and treatments for this devastating degenerative disorder. The present models using fruit flies, worms, rodents, non-human primates and induced pluripotent stem cells have helped researchers understand important disease-related mechanisms and test potential therapeutic options. However, the challenge of creating an ideal model still looms, for these models did not recapitulates all symptoms, particularly neurological presentation, of ATTR amyloidosis. Recently, knock-in techniques was used to generate two humanized ATTR mouse models, leading to amyloid deposition in the nerves and neuropathic manifestation in these models. This review gives a recent update on the milestone, progress, and challenges in developing different models for ATTR amyloidosis research. |
| first_indexed | 2024-04-14T05:42:01Z |
| format | Article |
| id | doaj.art-49f484de64394bc5ae95880dad011bb3 |
| institution | Directory Open Access Journal |
| issn | 1664-042X |
| language | English |
| last_indexed | 2024-04-14T05:42:01Z |
| publishDate | 2019-04-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Physiology |
| spelling | doaj.art-49f484de64394bc5ae95880dad011bb32022-12-22T02:09:24ZengFrontiers Media S.A.Frontiers in Physiology1664-042X2019-04-011010.3389/fphys.2019.00338441908Contributions of Animal Models to the Mechanisms and Therapies of Transthyretin AmyloidosisRidwan Babatunde Ibrahim0Ridwan Babatunde Ibrahim1Yo-Tsen Liu2Yo-Tsen Liu3Yo-Tsen Liu4Yo-Tsen Liu5Ssu-Yu Yeh6Jin-Wu Tsai7Jin-Wu Tsai8Institute of Brain Science, School of Medicine, National Yang-Ming University, Taipei, TaiwanTaiwan International Graduate Program in Interdisciplinary Neuroscience, National Yang-Ming University and Academia Sinica, Taipei, TaiwanInstitute of Brain Science, School of Medicine, National Yang-Ming University, Taipei, TaiwanDepartment of Neurology, Neurological Institute, Taipei Veterans General Hospital, Taipei, TaiwanDepartment of Medicine, School of Medicine, National Yang-Ming University, Taipei, TaiwanBrain Research Center and Biophotonics and Molecular Imaging Research Center, National Yang-Ming University, Taipei, TaiwanInstitute of Brain Science, School of Medicine, National Yang-Ming University, Taipei, TaiwanInstitute of Brain Science, School of Medicine, National Yang-Ming University, Taipei, TaiwanBrain Research Center and Biophotonics and Molecular Imaging Research Center, National Yang-Ming University, Taipei, TaiwanTransthyretin amyloidosis (ATTR amyloidosis) is a fatal systemic disease caused by amyloid deposits of misfolded transthyretin, leading to familial amyloid polyneuropathy and/or cardiomyopathy, or a rare oculoleptomeningeal amyloidosis. A good model system that mimic the disease phenotype is crucial for the development of drugs and treatments for this devastating degenerative disorder. The present models using fruit flies, worms, rodents, non-human primates and induced pluripotent stem cells have helped researchers understand important disease-related mechanisms and test potential therapeutic options. However, the challenge of creating an ideal model still looms, for these models did not recapitulates all symptoms, particularly neurological presentation, of ATTR amyloidosis. Recently, knock-in techniques was used to generate two humanized ATTR mouse models, leading to amyloid deposition in the nerves and neuropathic manifestation in these models. This review gives a recent update on the milestone, progress, and challenges in developing different models for ATTR amyloidosis research.https://www.frontiersin.org/article/10.3389/fphys.2019.00338/fulltransthyretinfamilial amyloid polyneuropathyC. elegansD. melanogastertransgenic mouseiPSC |
| spellingShingle | Ridwan Babatunde Ibrahim Ridwan Babatunde Ibrahim Yo-Tsen Liu Yo-Tsen Liu Yo-Tsen Liu Yo-Tsen Liu Ssu-Yu Yeh Jin-Wu Tsai Jin-Wu Tsai Contributions of Animal Models to the Mechanisms and Therapies of Transthyretin Amyloidosis Frontiers in Physiology transthyretin familial amyloid polyneuropathy C. elegans D. melanogaster transgenic mouse iPSC |
| title | Contributions of Animal Models to the Mechanisms and Therapies of Transthyretin Amyloidosis |
| title_full | Contributions of Animal Models to the Mechanisms and Therapies of Transthyretin Amyloidosis |
| title_fullStr | Contributions of Animal Models to the Mechanisms and Therapies of Transthyretin Amyloidosis |
| title_full_unstemmed | Contributions of Animal Models to the Mechanisms and Therapies of Transthyretin Amyloidosis |
| title_short | Contributions of Animal Models to the Mechanisms and Therapies of Transthyretin Amyloidosis |
| title_sort | contributions of animal models to the mechanisms and therapies of transthyretin amyloidosis |
| topic | transthyretin familial amyloid polyneuropathy C. elegans D. melanogaster transgenic mouse iPSC |
| url | https://www.frontiersin.org/article/10.3389/fphys.2019.00338/full |
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