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Safer conditioning for blood stem cell transplants

Nature Biotechnology volume 34, pages 721–723 (2016)Cite this article

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An antibody-based conditioning agent may open the way to wider application of gene therapy with hematopoietic stem cells.

The main applications of HSCT today are in the treatment of hematopoietic malignancies, like leukemias and lymphomas; of several inherited diseases of the hematopoietic system, including primary immunodeficiencies, thalassemia and sickle-cell disease; and of severe metabolic disorders such as lysosomal storage diseases. Before a stem cell transplant, patients undergo a conditioning regimen to deplete highly proliferating hematopoietic progenitors from the bone marrow and promote engraftment of donor HSCs. Because recipient and donor cells often have some degree of immunological mismatch that may lead to graft rejection or graft-versus-host disease, conditioning includes immunosuppressive drugs. For neoplastic disorders, preparatory regimens are also meant to reduce the tumor burden.

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Figure 1: A comparison of conventional allogeneic HSCT with autologous HSCT using genetically corrected or uncorrected stem cells.

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Authors and Affiliations

  1. Alessandro Aiuti and Luigi Naldini are at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Milan, Italy and Vita Salute San Raffaele University, Milan, Italy.,

    Alessandro Aiuti & Luigi Naldini

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  1. Alessandro Aiuti
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  2. Luigi Naldini
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Correspondence to Alessandro Aiuti or Luigi Naldini.

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Aiuti, A., Naldini, L. Safer conditioning for blood stem cell transplants. Nat Biotechnol 34, 721–723 (2016). https://doi.org/10.1038/nbt.3629

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