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Cell & Gene Therapy Insights

Cell & Gene Therapy Insights

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Towards individualized, low toxic conditioning in autologous gene-transduced hematopoietic cell transplantation

Clinical Trial Designs For Advanced Therapies

Rick Admiraal, Susan Prockop & Jaap Jan Boelens

Allogeneic hematopoietic cell transplantation (Allo-HCT) has become much safer over the last couple of years. This, together with the rapidly evolving autologous HCT-gene therapy options, is expected to result in an increase in the number of patients receiving HCT. Autologous HCT-gene therapy is a more advanced, safer and precise option for monogenetic life threatening disorders. The efficacy of gene therapy, however, does not only rely on the gene construct itself, but also on the conditioning applied before the gene therapy. In this review we describe how the conditioning can impact the outcomes of the allo-HCT and gene therapy and we will provide a future perspective on how to further improve the efficacy and reduce the short- and long-term toxicity of the conditioning.

DOI: 10.18609/cgti.2019.155
Submitted for review: October 2, 2019
Citation: Cell & Gene Therapy Insights 2019; 5(11), 1495-1503.
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