期刊
JOURNAL OF CLINICAL INVESTIGATION
卷 117, 期 6, 页码 1456-1465出版社
AMER SOC CLINICAL INVESTIGATION INC
DOI: 10.1172/JCI30953
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资金
- Telethon [GAT0203] Funding Source: Medline
Inherited and acquired diseases of the hematopoietic system can be cured by allogeneic hematopoietic stem cell transplantation. This treatment strategy is highly successful when an HLA-matched sibling donor is available, but if not, few therapeutic options exist. Gene-modified, autologous bone marrow transplantation can circumvent the severe immunological complications that occur when a related HLA-mismatched donor is used and thus represents an attractive alternative. in therapy this review, we summarize the advantages and limitations associated with the use of gene to cure SCID. Insertional mutagenesis and technological improvements aimed at increasing the safety of this strategy are also discussed.
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