Gene therapy and genome editing Journal Article


Authors: Boulad, F.; Mansilla-Soto, J.; Cabriolu, A.; Rivière, I.; Sadelain, M.
Article Title: Gene therapy and genome editing
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. © 2018 Elsevier Inc.
Keywords: gene transfer; lentivirus; thalassemia; crispr/cas9; gene editing
Journal Title: Hematology/Oncology Clinics of North America
Volume: 32
Issue: 2
ISSN: 0889-8588
Publisher: Elsevier Inc.  
Date Published: 2018-04-01
Start Page: 329
End Page: 342
Language: English
DOI: 10.1016/j.hoc.2017.11.007
PROVIDER: scopus
PUBMED: 29458735
DOI/URL:
Notes: Review -- Export Date: 1 March 2018 -- Source: Scopus
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MSK Authors
  1. Farid Boulad
    254 Boulad
  2. Michel W J Sadelain
    456 Sadelain
  3. Isabelle C Riviere
    164 Riviere