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Restoration of Human B-cell Differentiation Into NOD-SCID Mice Engrafted With Gene-corrected CD34+ Cells Isolated From Artemis or RAG1-deficient Patients

25 Jan 2008

Abstract excerpt

Severe combined immunodeficiency (SCID) caused by mutation of the recombination-activating gene 1 (RAG1) or Artemis gene lead to the absence of B- and T-cell differentiation. The only curative treatment is allogeneic bone marrow (BM) transplantation, which displays a high survival rate when an HLA compatible donor is available but has a poorer prognosis when the donor is partially compatible. Consequently, gene...

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PMID: 18223550
DOI: 10.1038/sj.mt.6300353

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