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Michael Triebwasser

Children's Hospital of Philadelphia

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A Severe Mouse Model of Alpha-Thalassemia to Study Abnormal Iron Metabolism and Erythropoiesis, Hematopoietic Stem Cell Behavior and Development of a Gene Therapy Approach for Its Treatment

2021 Blood
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Improved Gene Therapy for Metachromatic Leukodystrophy

2021 Blood
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