From Mutation to Regeneration: Stem Cell Strategies for Treating Genetic Disorders

Genetic disorders, which arise from hereditary mutations, frequently lead to chronic or potentially fatal health issues with limited therapeutic alternatives. Stem cell therapy presents a promising approach by facilitating the regeneration of damaged tissues and the rectification of genetic anomalies. This review examines contemporary strategies employing hematopoietic, mesenchymal, and induced pluripotent stem cells (iPSCs) for the treatment of genetic disorders. The incorporation of gene editing technologies, such as CRISPR-Cas9, has broadened the scope of therapeutic interventions, especially for monogenic disorders, including sickle cell disease, β-thalassemia, and severe combined immunodeficiency. Additionally, the review addresses emerging applications of these therapies in the context of neurodegenerative and metabolic diseases. Despite persistent challenges concerning safety, delivery mechanisms, and the long-term effectiveness of these treatments, advancements in stem cell-based therapies are progressing swiftly toward clinical implementation. These developments signify a pivotal transition in the realm of genetic medicine, moving from mere symptom management to the prospect of potentially curative interventions.