# Progress of iPSC-derived retinal organoids in the study of inherited retinal diseases

## TL;DR

This paper reviews how retinal organoids derived from stem cells help study and treat inherited eye diseases.

## Contribution

The paper systematically reviews the use of iPSC-derived retinal organoids for modeling specific inherited retinal diseases.

## Key findings

- iPSC-derived retinal organoids are effective models for studying IRDs like retinoblastoma and retinitis pigmentosa.
- ROs mimic in-vivo retinal development through structural formation and physiological functions.
- ROs enable drug screening and pathogenesis exploration for improved precision treatment of IRDs.

## Abstract

Inherited retinal diseases (IRDs) constitute a complex and heterogeneous group of rare disorders characterized by significant genetic diversity. These conditions often lead to the degeneration of photoreceptor cells, resulting in severe visual impairment. A major challenge in the study and treatment of IRDs is the lack of appropriate preclinical models for investigating their pathogenesis and evaluating potential therapeutic interventions. In recent years, advances in retinal organoids (ROs) culture technology have provided promising new avenues for IRDs. This review systematically elaborates on the applications of induced pluripotent stem cells (iPSC)-derived ROs as disease models in IRDs such as retinoblastoma (RB), retinitis pigmentosa (RP), Leber congenital amaurosis (LCA), and X-linked juvenile retinoschisis (XLRS). This review also briefly explores the culturing methods of iPSC-derived ROs in recent years. Specifically, it emphasizes the comparison of the similarities between the formation process of ROs and the in-vivo retinal development process. This encompasses aspects such as the formation of different structures, the expression of key markers, and the manifestation of physiological functions. By utilizing the RO disease model to explore the pathogenesis of IRDs and conduct drug screening, it is expected to advance the precision treatment of IRDs and improve the therapeutic outcomes for patients.