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In Utero Hematopoietic Stem Cell Transplantation for Congenital Disorders

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Abstract

Recent advances in stem cell biology, prenatal diagnosis, and fetal surgery have transformed our ability to use in utero stem cell transplantation to cure congenital anomalies. With the recent discovery of inducible pluripotent stem (iPS) cells [1], new opportunities are available to generate patient-matched iPS cells for specific diseases. Advances in prenatal imaging and molecular diagnostics allow us to accurately diagnose congenital hematologic diseases as early as 10–12 weeks of gestation [2]. Furthermore, high-resolution ultrasonography has made it technically feasible to deliver stem cells in the early gestation fetus. Fetal intervention in patients has expanded since its first description in 1982 [3] and is used to treat anatomic anomalies with both conventional and minimally invasive techniques [4]. The improved understanding of stem cell biology, the ability to diagnose congenital diseases that are amenable to prenatal therapy, and the technical capability to deliver cells safely in utero have brought renewed interest and excitement for the promise of prenatal stem cell therapy.

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Authors and Affiliations

  1. Eli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research, University of California, San Francisco, CA, USA

    Amar Nijagal M.D.

  2. Department of Surgery, University of California, 513 Parnassus Avenue HSW 1601, Campus, 0570, San Francisco, CA, 94143-0570, USA

    Tippi C. MacKenzie M.D.

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  1. Amar Nijagal M.D.
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  2. Tippi C. MacKenzie M.D.
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Correspondence to Tippi C. MacKenzie M.D. .

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  1. Southend Park 55, Kolkata, 700029, India

    Niranjan Bhattacharya

  2. Dept. Obstetrics & Gynaecology, Boston University Medical Centre, E. Concord Street 85, Boston, 02118, Massachusetts, USA

    Phillip Stubblefield

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Nijagal, A., MacKenzie, T.C. (2013). In Utero Hematopoietic Stem Cell Transplantation for Congenital Disorders. In: Bhattacharya, N., Stubblefield, P. (eds) Human Fetal Tissue Transplantation. Springer, London. https://doi.org/10.1007/978-1-4471-4171-6_12

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  • DOI: https://doi.org/10.1007/978-1-4471-4171-6_12

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