Gene Therapy and Genome Editing

Publication date: Available online 9 January 2018
Source:Hematology/Oncology Clinics of North America
Author(s): Farid Boulad, Jorge Mansilla-Soto, Annalisa Cabriolu, Isabelle Rivière, Michel Sadelain

Teaser

The β-thalassemias are inherited blood disorders that result from insufficient production of the β-chain of hemoglobin. More than 200 different mutations have been identified. β-Thalassemia major requires life-long transfusions. The only cure for severe β-thalassemia is to provide patients with hematopoietic stem cells. Globin gene therapy promises a curative autologous stem cell transplantation without the immunologic complications of allogeneic transplantation. The future directions of gene therapy include enhancement of lentiviral vector-based approaches, fine tuning of the conditioning regimen, and the design of safer vectors. Progress in genetic engineering bodes well for finding a cure for severe globin disorders.


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