Application of patient-derived induced pluripotent stem cells and organoids in inherited retinal diseases
Abstract Inherited retinal diseases (IRDs) can induce severe sight-threatening retinal degeneration and impose a considerable economic burden on patients and society, making efforts to cure blindness imperative. Transgenic animals mimicking human genetic diseases have long been used as a primary res...
| Main Authors: | , , , , |
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| Format: | Article |
| Language: | English |
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BMC
2023-11-01
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| Series: | Stem Cell Research & Therapy |
| Subjects: | |
| Online Access: | https://doi.org/10.1186/s13287-023-03564-5 |
| _version_ | 1827604176565174272 |
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| author | Yuqin Liang Xihao Sun Chunwen Duan Shibo Tang Jiansu Chen |
| author_facet | Yuqin Liang Xihao Sun Chunwen Duan Shibo Tang Jiansu Chen |
| author_sort | Yuqin Liang |
| collection | DOAJ |
| description | Abstract Inherited retinal diseases (IRDs) can induce severe sight-threatening retinal degeneration and impose a considerable economic burden on patients and society, making efforts to cure blindness imperative. Transgenic animals mimicking human genetic diseases have long been used as a primary research tool to decipher the underlying pathogenesis, but there are still some obvious limitations. As an alternative strategy, patient-derived induced pluripotent stem cells (iPSCs), particularly three-dimensional (3D) organoid technology, are considered a promising platform for modeling different forms of IRDs, including retinitis pigmentosa, Leber congenital amaurosis, X-linked recessive retinoschisis, Batten disease, achromatopsia, and best vitelliform macular dystrophy. Here, this paper focuses on the status of patient-derived iPSCs and organoids in IRDs in recent years concerning disease modeling and therapeutic exploration, along with potential challenges for translating laboratory research to clinical application. Finally, the importance of human iPSCs and organoids in combination with emerging technologies such as multi-omics integration analysis, 3D bioprinting, or microfluidic chip platform are highlighted. Patient-derived retinal organoids may be a preferred choice for more accurately uncovering the mechanisms of human retinal diseases and will contribute to clinical practice. |
| first_indexed | 2024-03-09T05:55:21Z |
| format | Article |
| id | doaj.art-73c6d2f55c1a4bfe838a19bd77287ba5 |
| institution | Directory Open Access Journal |
| issn | 1757-6512 |
| language | English |
| last_indexed | 2024-03-09T05:55:21Z |
| publishDate | 2023-11-01 |
| publisher | BMC |
| record_format | Article |
| series | Stem Cell Research & Therapy |
| spelling | doaj.art-73c6d2f55c1a4bfe838a19bd77287ba52023-12-03T12:14:17ZengBMCStem Cell Research & Therapy1757-65122023-11-0114111710.1186/s13287-023-03564-5Application of patient-derived induced pluripotent stem cells and organoids in inherited retinal diseasesYuqin Liang0Xihao Sun1Chunwen Duan2Shibo Tang3Jiansu Chen4Aier Eye InstituteAier Eye InstituteAier Eye InstituteAier Eye InstituteAier Eye InstituteAbstract Inherited retinal diseases (IRDs) can induce severe sight-threatening retinal degeneration and impose a considerable economic burden on patients and society, making efforts to cure blindness imperative. Transgenic animals mimicking human genetic diseases have long been used as a primary research tool to decipher the underlying pathogenesis, but there are still some obvious limitations. As an alternative strategy, patient-derived induced pluripotent stem cells (iPSCs), particularly three-dimensional (3D) organoid technology, are considered a promising platform for modeling different forms of IRDs, including retinitis pigmentosa, Leber congenital amaurosis, X-linked recessive retinoschisis, Batten disease, achromatopsia, and best vitelliform macular dystrophy. Here, this paper focuses on the status of patient-derived iPSCs and organoids in IRDs in recent years concerning disease modeling and therapeutic exploration, along with potential challenges for translating laboratory research to clinical application. Finally, the importance of human iPSCs and organoids in combination with emerging technologies such as multi-omics integration analysis, 3D bioprinting, or microfluidic chip platform are highlighted. Patient-derived retinal organoids may be a preferred choice for more accurately uncovering the mechanisms of human retinal diseases and will contribute to clinical practice.https://doi.org/10.1186/s13287-023-03564-5Retinal organoidInduced pluripotent stem cellInherited retinal diseaseDisease modelingTissue engineering |
| spellingShingle | Yuqin Liang Xihao Sun Chunwen Duan Shibo Tang Jiansu Chen Application of patient-derived induced pluripotent stem cells and organoids in inherited retinal diseases Stem Cell Research & Therapy Retinal organoid Induced pluripotent stem cell Inherited retinal disease Disease modeling Tissue engineering |
| title | Application of patient-derived induced pluripotent stem cells and organoids in inherited retinal diseases |
| title_full | Application of patient-derived induced pluripotent stem cells and organoids in inherited retinal diseases |
| title_fullStr | Application of patient-derived induced pluripotent stem cells and organoids in inherited retinal diseases |
| title_full_unstemmed | Application of patient-derived induced pluripotent stem cells and organoids in inherited retinal diseases |
| title_short | Application of patient-derived induced pluripotent stem cells and organoids in inherited retinal diseases |
| title_sort | application of patient derived induced pluripotent stem cells and organoids in inherited retinal diseases |
| topic | Retinal organoid Induced pluripotent stem cell Inherited retinal disease Disease modeling Tissue engineering |
| url | https://doi.org/10.1186/s13287-023-03564-5 |
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