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Preemptive therapy for genetic disease

Nature Medicinevolume 2pages161162 (1996) | Download Citation


In utero stem cell transplantation for inherited blood cell disorders.

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  1. 1

    Flake, A.W. et al. Successful treatment of X-linked recessive severe combined immunodeficiency (X-SCID) by the in utero transplantation of CD34 enriched paternal bone marrow cells. Blood 86, 125a (1995).

  2. 2

    Buckley, R.H. et al. Development of immunity in human severe primary T cell deficiency following haploidentical bone marrow stem cell transplantation. J. Immunoi. 136, 2398–2407 (1986).

  3. 3

    Noguchi, M. et al. Interleukin-2 receptor γ chain mutation results in X-linked severe combined immunodeficiency in humans. Cell 73, 147–157 (1993).

  4. 4

    Cowan, M.J. and Globus, M. In utero hematopoietic stem cell transplants for inherited diseases. Am. J. Pediat. Hematol. J. Oncol. 16, 35–42 (1994).

  5. 5

    Nesci, S. et al. Mixed chimerism in thalassemic patients after bone marrow transplantation. B.M.T. 10, 143–146 (1992).

  6. 6

    Kapelushnik, J. et al. Analysis of β-globin mutations shows stable mixed chimerism in patients with thalassemia after bone marrow transplantation. Blood 86, 3241–3246 (1995).

  7. 7

    Lucarelli, G. et al. Marrow transplantation in patients with thalassemia responsive to chelation therapy N. Engl. J. Med. 329, 840–844 (1993).

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  1. Divisions of Hematology and Medical Genetics, University of Washington, Seattle, Washington, 98195, USA

    • C.Anthony Blau
    •  & George Stamatoyannopoulos


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