Gene therapy for transfusion-dependent beta thalassemia patients?

Can gene therapy eliminate the need for blood transfusions for patients with transfusion-dependent β thalassemia?

This article reports the results of two phase 1-2 studies studied on the use of a gene therapy.  In these studies, patients with transfusion-depenent β-thalassemia received gene therapy to assess if this therapy would substitute for the standard therapy, red-cell blood transfusions.  The researchers were assessing both the safety and the efficacy of this gene therapy.  You can read the results of this study in the New England Journal of Medicine.

Thompson, A. A., Walters, M. C., Kwiatkowski, J., Rasko, J. E., Ribeil, J. A., Hongeng, S., … & Moshous, D. (2018). Gene therapy in patients with transfusion-dependent β-thalassemiaNew England Journal of Medicine378(16), 1479-1493.

To learn more about this area of gene therapy, explore additional readings from Himmelfarb Library’s full-text collection:

  • Boulad F, Mansilla-Soto J, Cabriolu A, Rivière I, Sadelain M. 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. Review. PubMed PMID: 29458735.
  • Ferrari G, Cavazzana M, Mavilio F. Gene Therapy Approaches to
    Hemoglobinopathies
    . Hematol Oncol Clin North Am. 2017 Oct;31(5):835-852. doi:
    10.1016/j.hoc.2017.06.010. Review. PubMed PMID: 28895851.
  • Glaser A, McColl B, Vadolas J. The therapeutic potential of genome editing for
    β-thalassemia
    . F1000Res. 2015 Dec 11;4. pii: F1000 Faculty Rev-1431. doi:
    10.12688/f1000research.7087.1. eCollection 2015. Review. PubMed PMID: 26918126;
    PubMed Central PMCID: PMC4753996.

Explore more of Himmelfarb Library’s genetics collection by checking out:

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