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Gene Therapies for Transfusion-Dependent b-Thalassemia

Sandeep Soni1

  • 1Division of Pediatric Bone Marrow Transplant, Department of Pediatrics, University of California, San Francisco, CA, USA. Correspondence to: Dr Sandeep Soni, Associate Clinical Professor, Division of Pediatric Bone Marrow Transplant, Department of Pediatrics, University of California, San Francisco, CA, 94158, USA. sandeep.soni@ucsf.edu.

Indian Pediatrics
|March 27, 2021
PubMed
Summary

Gene therapy offers a promising new treatment for beta-thalassemia, a severe anemia caused by genetic defects. Advances in gene editing and stem cell manipulation are paving the way for effective therapies, especially for patients without transplant donors.

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