Gene Therapy and Gene Editing

Innovative Curative Treatment of Beta-Thalassemia: Cost-efficacy Analysis of Gene Therapy Versus Allogenic Hematopoietic Stem Cell Transplantation

Seventy-five percent of patients with beta thalassemia (β-thalassemia) do not have human leukocyte antigen–matched siblings and until recently had no access to a curative treatment. Gene therapy is a promising…

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