Abstract:
:Severe combined immunodeficiency (SCID) conditions appear to be the best possible candidates for a gene therapy approach. Transgene expression by lymphocyte precursors should confer to these cells a selective growth advantage that gives rise to long-lived T-lymphocytes. This rationale was used as a basis for a clinical trial of the SCID-X1 disorder caused by common gamma (gamma c) gene mutations. This trial consists of ex vivo retroviral-mediated (MFG-B2 gamma c vector) gammac gene transfer into marrow CD34+ cells in CH-296 fibronectin fragment-coated bags. Up to now, 9 patients with typical SCID-X1 diagnosed within the first year of life and lacking an HLA-identical donor have been enrolled. More than 2 years' assessment of 5 patients and more than 1 year for 7 patients provide evidence for full development of functional, mature T-cells in the absence of any adverse effects. Functional transduced natural killer cells are also detectable, although in low numbers. All but 1 patient with T-cell immunity have also developed immunoglobulin production, which has alleviated the need for intravenous immunoglobulin substitution despite a low detection frequency of transduced B-cells. These 8 patients are doing well and living in a normal environment. This yet successful gene therapy demonstrates that in a setting where transgene expression provides a selective advantage, a clinical benefit can be expected.
journal_name
Int J Hematoljournal_title
International journal of hematologyauthors
Hacein-Bey-Abina S,Fischer A,Cavazzana-Calvo Mdoi
10.1007/BF02982686subject
Has Abstractpub_date
2002-11-01 00:00:00pages
295-8issue
4eissn
0925-5710issn
1865-3774journal_volume
76pub_type
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journal_title:International journal of hematology
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journal_title:International journal of hematology
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pub_type: 杂志文章
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journal_title:International journal of hematology
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更新日期:2000-02-01 00:00:00
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journal_title:International journal of hematology
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pub_type: 临床试验,杂志文章,多中心研究
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