Patient-derived induced pluripotent stem cell organoids for amyotrophic lateral sclerosis drug discovery
Complex biological mechanisms and unidentified therapeutic targets for amyotrophic lateral sclerosis (ALS) significantly hinder the development of effective treatments. Given these challenges, reliable disease models that accurately replicate ALS phenotypes with relevant biological underpinnings are...
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| Main Authors: | , , , , , , |
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| Format: | Article |
| Language: | English |
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Compuscript Ltd
2025-01-01
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| Series: | Acta Materia Medica |
| Online Access: | https://www.scienceopen.com/hosted-document?doi=10.15212/AMM-2024-0077 |
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| _version_ | 1850121605827526656 |
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| author | Wenyan Li Jingqi Liu Wenting Li Lina Sun Hao Zhang Lei Gao Chong Gao |
| author_facet | Wenyan Li Jingqi Liu Wenting Li Lina Sun Hao Zhang Lei Gao Chong Gao |
| author_sort | Wenyan Li |
| collection | DOAJ |
| description | Complex biological mechanisms and unidentified therapeutic targets for amyotrophic lateral sclerosis (ALS) significantly hinder the development of effective treatments. Given these challenges, reliable disease models that accurately replicate ALS phenotypes with relevant biological underpinnings are essential for advancing precision medicine in ALS. Patient-derived induced pluripotent stem cell (iPSC) organoids have emerged as an innovative tool for disease modeling and drug evaluation. Growing evidence highlights the advantages of organoids in replicating ALS phenotypes and supporting drug development. However, challenges remain in utilizing organoids for ALS drug testing and other neurodegenerative diseases. In this review we summarize the current progress in ALS model development, encompassing both in vitro and in vivo non-human models, as well as iPSC-derived human models. Furthermore, within the context of ALS drug screening, we discuss critical considerations for applying organoids to evaluate disease-associated phenotypes and to accurately reflect disease-related symptoms. |
| format | Article |
| id | doaj-art-e72809d1f8b74618ae5ff4609a646f3c |
| institution | OA Journals |
| issn | 2737-7946 |
| language | English |
| publishDate | 2025-01-01 |
| publisher | Compuscript Ltd |
| record_format | Article |
| series | Acta Materia Medica |
| spelling | doaj-art-e72809d1f8b74618ae5ff4609a646f3c2025-08-20T02:35:03ZengCompuscript LtdActa Materia Medica2737-79462025-01-0141365010.15212/AMM-2024-0077Patient-derived induced pluripotent stem cell organoids for amyotrophic lateral sclerosis drug discoveryWenyan LiJingqi LiuWenting LiLina SunHao ZhangLei GaoChong GaoComplex biological mechanisms and unidentified therapeutic targets for amyotrophic lateral sclerosis (ALS) significantly hinder the development of effective treatments. Given these challenges, reliable disease models that accurately replicate ALS phenotypes with relevant biological underpinnings are essential for advancing precision medicine in ALS. Patient-derived induced pluripotent stem cell (iPSC) organoids have emerged as an innovative tool for disease modeling and drug evaluation. Growing evidence highlights the advantages of organoids in replicating ALS phenotypes and supporting drug development. However, challenges remain in utilizing organoids for ALS drug testing and other neurodegenerative diseases. In this review we summarize the current progress in ALS model development, encompassing both in vitro and in vivo non-human models, as well as iPSC-derived human models. Furthermore, within the context of ALS drug screening, we discuss critical considerations for applying organoids to evaluate disease-associated phenotypes and to accurately reflect disease-related symptoms.https://www.scienceopen.com/hosted-document?doi=10.15212/AMM-2024-0077 |
| spellingShingle | Wenyan Li Jingqi Liu Wenting Li Lina Sun Hao Zhang Lei Gao Chong Gao Patient-derived induced pluripotent stem cell organoids for amyotrophic lateral sclerosis drug discovery Acta Materia Medica |
| title | Patient-derived induced pluripotent stem cell organoids for amyotrophic lateral sclerosis drug discovery |
| title_full | Patient-derived induced pluripotent stem cell organoids for amyotrophic lateral sclerosis drug discovery |
| title_fullStr | Patient-derived induced pluripotent stem cell organoids for amyotrophic lateral sclerosis drug discovery |
| title_full_unstemmed | Patient-derived induced pluripotent stem cell organoids for amyotrophic lateral sclerosis drug discovery |
| title_short | Patient-derived induced pluripotent stem cell organoids for amyotrophic lateral sclerosis drug discovery |
| title_sort | patient derived induced pluripotent stem cell organoids for amyotrophic lateral sclerosis drug discovery |
| url | https://www.scienceopen.com/hosted-document?doi=10.15212/AMM-2024-0077 |
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