Article

Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia: Update from the Northstar Hgb-204 Phase 1/2 Clinical Study

2016-12-02

Abstract excerpt

Abstract BACKGROUND Allogeneic hematopoietic stem cell (HSC) transplant is potentially curative for patients with β-thalassemia major or, as more broadly defined, transfusion dependent β-thalassemia (TDT). However, HSC transplant is generally restricted to younger patients with matched sibling donors. Gene therapy could provide a transformative treatment for a broader population of patients with TDT, including tho...

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Literature Corpus work: 7d3520cc-7c36-526d-84b8-21dbedf2ada8
DOI: 10.1182/blood.v128.22.1175.1175

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