Story perspectives

UCLA Team Develops Promising Stem Cell Therapy for Thalassemia

9/18/2025

42 10

Summary

Story summary

UCLA researchers are developing a stem cell gene therapy for alpha thalassemia major, a severe blood disorder.
The therapy utilizes a patient's blood stem cells to introduce the missing alpha-globin gene using a viral vector.
Preclinical studies successfully restored normal hemoglobin production in patient cells.
Led by Dr. Donald Kohn, the team aims for a one-time curative treatment.
Funding from the California Institute for Regenerative Medicine will support further studies and clinical trials.

1 of 1

Story summary

UCLA researchers are developing a stem cell gene therapy for alpha thalassemia major, a severe blood disorder.
The therapy utilizes a patient's blood stem cells to introduce the missing alpha-globin gene using a viral vector.
Preclinical studies successfully restored normal hemoglobin production in patient cells.
Led by Dr. Donald Kohn, the team aims for a one-time curative treatment.
Funding from the California Institute for Regenerative Medicine will support further studies and clinical trials.