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Updated: Jun 23, 2025

In Situ Immunofluorescent Staining of Autophagy in Muscle Stem Cells
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Therapeutic Relevance of Inducing Autophagy in β-Thalassemia

Roberto Gambari1, Alessia Finotti1

  • 1Center "Chiara Gemmo and Elio Zago" for the Research on Thalassemia, Department of Life Sciences and Biotechnology, University of Ferrara, 44121 Ferrara, Italy.

Cells
|June 19, 2024
PubMed

The β-thalassemias are inherited genetic disorders affecting the hematopoietic system. In β-thalassemias, more than 350 mutations of the adult β-globin gene cause the low or absent production of adult hemoglobin (HbA). A clinical parameter affecting the physiology of erythroid cells is the excess of free α-globin. Possible experimental strategies for a reduction in excess free α-globin chains in β-thalassemia are CRISPR-Cas9-based genome editing of the β-globin gene, forcing "de novo" HbA production and fetal hemoglobin (HbF) induction. In addition, a reduction in excess free α-globin chains in β-thalassemia can be achieved by induction of the autophagic process. This process is regulated by the Unc-51-like kinase 1 (Ulk1) gene. The interplay with the PI3K/Akt/TOR pathway, with the activity of the α-globin stabilizing protein (AHSP) and the involvement of microRNAs in autophagy and Ulk1 gene expression, is presented and discussed in the context of identifying novel biomarkers and potential therapeutic targets for β-thalassemia.

Keywords:
Ulk1autophagyineffective erythropoiesisα-globinβ-thalassemia

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