Application of patient-derived induced pluripotent stem cells and organoids in inherited retinal diseases.
Stem Cell Res Ther
; 14(1): 340, 2023 11 27.
Article
en En
| MEDLINE
| ID: mdl-38012786
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.
Palabras clave
Texto completo:
1
Banco de datos:
MEDLINE
Asunto principal:
Degeneración Retiniana
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Retinitis Pigmentosa
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Células Madre Pluripotentes Inducidas
Límite:
Animals
/
Humans
Idioma:
En
Revista:
Stem Cell Res Ther
Año:
2023
Tipo del documento:
Article
País de afiliación:
China