Bombieri, Cristina, Corsi, Andrea, Trabetti, Elisabetta, Ruggiero, Alessandra 
ORCID: 0000-0001-6436-8814, Marchetto, Giulia, Vattemi, Gaetano, Valenti, Maria Teresa, Zipeto, Donato and Romanelli, Maria Grazia
(2024)
Advanced Cellular Models for Rare Disease Study: Exploring Neural, Muscle and Skeletal Organoids.
International journal of molecular sciences, 25 (2).
p. 1014.
Abstract
Organoids are self-organized, three-dimensional structures derived from stem cells that can mimic the structure and physiology of human organs. Patient-specific induced pluripotent stem cells (iPSCs) and 3D organoid model systems allow cells to be analyzed in a controlled environment to simulate the characteristics of a given disease by modeling the underlying pathophysiology. The recent development of 3D cell models has offered the scientific community an exceptionally valuable tool in the study of rare diseases, overcoming the limited availability of biological samples and the limitations of animal models. This review provides an overview of iPSC models and genetic engineering techniques used to develop organoids. In particular, some of the models applied to the study of rare neuronal, muscular and skeletal diseases are described. Furthermore, the limitations and potential of developing new therapeutic approaches are discussed.
| Item Type: | Article |
|---|---|
| Uncontrolled Keywords: | Muscles, Organoids, Animals, Humans, Rare Diseases, Genetic Engineering, Induced Pluripotent Stem Cells |
| Divisions: | Faculty of Health and Life Sciences Faculty of Health and Life Sciences > Institute of Systems, Molecular and Integrative Biology |
| Depositing User: | Symplectic Admin |
| Date Deposited: | 14 Feb 2024 11:13 |
| Last Modified: | 26 Feb 2024 07:19 |
| DOI: | 10.3390/ijms25021014 |
| Open Access URL: | https://doi.org/10.3390/ijms25021014 |
| Related URLs: | |
| URI: | https://livrepository.liverpool.ac.uk/id/eprint/3178639 |
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