Biomedical Applications of Organoids in Genetic Diseases

Organoid technology, using 3D structures derived from stem cells, has advanced the study of genetic diseases by replicating human organ complexity and allowing for patient-specific genetic editing. This review highlights their application in modeling genetic disorders across various organs, aiding in personalized medicine and drug discovery. Organoids are particularly useful in diagnosing and treating conditions like cystic fibrosis and Mucin 1 kidney disease, and they facilitate personalized therapy testing, including CRISPR/Cas9 gene editing. Organoid transplantation shows promise in regenerative therapies, especially for retinal diseases. Despite challenges like vascularization and cost, organoids offer a promising platform for understanding disease mechanisms and developing targeted therapies, with ethical considerations and in vivo validation remaining crucial.