Gene Therapy and Genome Editing - 21/02/18
, Jorge Mansilla-Soto, PhD a, Annalisa Cabriolu, PhD a, Isabelle Rivière, PhD a, Michel Sadelain, MD, PhD aRésumé |
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.
Le texte complet de cet article est disponible en PDF.Keywords : Thalassemia, Gene transfer, Gene editing, Lentivirus, CRISPR/Cas9
Plan
| No conflicts of interest for all authors. |
Vol 32 - N° 2
P. 329-342 - avril 2018 Retour au numéro
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