Gene Therapy and Genome Editing

Farid Boulad; Jorge Mansilla-Soto; Annalisa Cabriolu; Isabelle Rivière; Michel Sadelain

doi:10.1016/j.hoc.2017.11.007

Gene Therapy and Genome Editing

Hematol Oncol Clin North Am. 2018 Apr;32(2):329-342. doi: 10.1016/j.hoc.2017.11.007. Epub 2018 Jan 9.

Authors

Farid Boulad¹, Jorge Mansilla-Soto², Annalisa Cabriolu², Isabelle Rivière², Michel Sadelain²

Affiliations

¹ Center for Cell Engineering, Memorial Sloan Kettering Cancer Center, 1275 York Avenue, New York, NY 10065, USA; Department of Pediatrics, Memorial Sloan Kettering Cancer Center, 1275 York Avenue, New York, NY 10065, USA. Electronic address: bouladf@mskcc.org.
² Center for Cell Engineering, Memorial Sloan Kettering Cancer Center, 1275 York Avenue, New York, NY 10065, USA.

PMID: 29458735
DOI: 10.1016/j.hoc.2017.11.007

Abstract

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.

Keywords: CRISPR/Cas9; Gene editing; Gene transfer; Lentivirus; Thalassemia.

Publication types

Review

MeSH terms

CRISPR-Cas Systems
Gene Editing* / methods
Gene Transfer Techniques
Genetic Therapy* / methods
Genetic Vectors / genetics
Humans
Transduction, Genetic
beta-Globins / genetics*
beta-Thalassemia / genetics*
beta-Thalassemia / therapy*

Substances

beta-Globins