Research Objective The objective of this research is to define the final therapeutic candidate for effective hematopoietic stem cell gene therapy to treat severe alpha thalassemia that requires life-ling transfusions…
Therapeutic Candidate or Device Maternal bone marrow-derived CD34+ hematopoietic stem cells. Indication Fetal alpha thalassemia major. Therapeutic Mechanism This strategy that takes advantage of existing tolerance between the mother and…
Dr. MacKenzie and her team at UCSF are using hematopoietic stem cells (HSCs) to treat babies in the womb who have alpha thalassemia major, a blood disorder that is almost always fatal. Current…
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