Modeling rare genetic skeletal disorders with bone organoids: a narrative review.
WHY IT MATTERS
For patients with rare skeletal disorders, bone organoids could speed up the discovery of treatments by allowing researchers to test drugs on disease-specific bone tissue grown from patient cells, potentially leading to personalized medicine approaches.
Scientists are developing a new way to study rare bone diseases using lab-grown bone tissue called organoids. Instead of only using animal tests or simple cell cultures, researchers can now grow tiny bone structures that act like real bones. This helps them understand why these diseases happen and test new treatments more accurately before trying them in patients.
Modeling rare genetic skeletal disorders with bone organoids: a narrative review. Abstract: Rare genetic skeletal disorders (RGSDs) encompass a heterogeneous group of hundreds rare conditions affecting the skeletal system. The rarity of these disorders, phenotypic and genetic diversity, combined with the limitations of conventional cellular and animal RGSD models, have hindered progress in understanding their pathophysiology and developing effective therapies. However, the latest advances in stem cell and bone tissue engineering techniques offer transformative opportunities in investigation of RGSD, particularly through bone organoids that enable disease modeling within a precision medicine framework. This review outlines the progress in RGSD organoid research, starting with the pivotal concepts of RGSDs bone biology, and extending to the disease-specific molecular signatures ess Authors: Micha et al. Journal: Bone MeSH: Humans, Organoids, Bone Diseases, Animals, Bone and Bones, Rare Diseases, Models, Biological, Tissue Engineering