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In Utero Hematopoietic Cell Transplantation: Past Clinical Experience and Future Clinical Trials

  • Prenatal Therapies (WH Peranteau, Section Editor)
  • Published:
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Abstract

Purpose of Review

In utero hematopoietic stem cell transplantation is an evolving therapy with possible implications for the treatment of many fetal disorders. Here, we present the past clinical experience and current clinical trials and discuss future directions for in utero therapy.

Recent Findings

Barriers to clinically meaningful in utero hematopoietic stem cell transplantation have been identified including maternal T cell-mediated graft rejection, achieving adequate space within the fetal bone marrow niche, and delivering adequate stem cells intravascularly. In a canine model of hematopoietic stem cell transplantation, high chimerism levels were achieved with maternal stem cells delivered intravascularly. Chimerism remained stable over a 2-year period without any evidence of graft-versus-host disease. Current clinical trials such as one for alpha thalassemia seek to apply these results in human diseases.

Summary

The field of in utero stem cell transplantation continues to improve and expand on the possible diseases that can be treated. The reasons for limited success in previous trials have been identified and current trials utilizing maternally derived stem cells at high doses are underway.

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Authors and Affiliations

  1. The Center for Maternal-Fetal Precision Medicine, University of California, San Francisco, CA, USA

    Russell G. Witt, Quoc-Hung L. Nguyen & Tippi C. MacKenzie

  2. The Department of Surgery, University of California, San Francisco, CA, USA

    Russell G. Witt, Quoc-Hung L. Nguyen & Tippi C. MacKenzie

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  1. Russell G. Witt
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  2. Quoc-Hung L. Nguyen
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  3. Tippi C. MacKenzie
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Correspondence to Tippi C. MacKenzie.

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Russell G. Witt, Quoc-Hung L. Nguyen, and Tippi C. MacKenzie declare that they have no conflict of interest.

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This article does not contain any studies with human or animal subjects performed by any of the authors.

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This article is part of the Topical Collection on Prenatal Therapies

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Witt, R.G., Nguyen, QH.L. & MacKenzie, T.C. In Utero Hematopoietic Cell Transplantation: Past Clinical Experience and Future Clinical Trials. Curr Stem Cell Rep 4, 74–80 (2018). https://doi.org/10.1007/s40778-018-0119-7

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  • DOI: https://doi.org/10.1007/s40778-018-0119-7

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