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- W2044532250 abstract "Recent clinical trials in patients with a severe combined immunodeficiency disease demonstrate that gene therapy is a powerful tool in the treatment of genetic blood defects. Recent identification of the genes involved in the pathogenesis of inherited lymphohemopoietic disorders led to animal models of gene transfer. Extensive preclinical studies have overcome some of the obstacles involved in the transduction of hemopoietic cells. These promising results led to the approval of several clinical trials that are currently underway. This review focuses on the clinical outcome in patients with genetic blood defects treated by gene transfer and examines the progress achieved to date and the problems that have been encountered. Despite the obstacles, improved clinical results for several of these diseases are expected within the next 5 years." @default.
- W2044532250 created "2016-06-24" @default.
- W2044532250 creator A5007387587 @default.
- W2044532250 creator A5088946190 @default.
- W2044532250 date "2001-11-01" @default.
- W2044532250 modified "2023-09-25" @default.
- W2044532250 title "Correction of genetic blood defects by gene transfer" @default.
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- W2044532250 doi "https://doi.org/10.1097/00062752-200111000-00008" @default.
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