| Author(s) | Nathwani AC; Benjamin R; Nienhuis AW; Davidoff AM
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| Title | Current status and prospects for gene therapy
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| Source | VOX SANGUINIS 87(2):73-81
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| Date | 2004 AUG
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| Type | Journal : Review
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| LCR: 3 NCR: 43 LCS: 0 GCS: 1
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| Address | Univ Coll London, Dept Haematol, London WC1E 6HX, England.
Natl Blood Serv, London, England.
St Jude Childrens Hosp, Div Expt Hematol, Memphis, TN 38105 USA.
St Jude Childrens Hosp, Dept Surg, Memphis, TN 38105 USA.
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| Reprint | Nathwani, AC, Univ Coll London, Dept Haematol, 99 Chenies Mews, London
WC1E 6HX, England.
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| E-mail | a.nathwani@ucl.ac.uk
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| Abstract | Gene therapy is a new and exciting therapeutic concept that offers the promise of cure for an array of inherited, malignant and infectious disorders. After years of failure, substantial progress in the efficiency of gene-transfer technology has recently resulted in impressive clinical success in infants with immunodeficiency. Two of these children have, however, subsequently developed leukaemia as a result of insertional mutagenesis, raising concerns about the safety of genetic therapeutics. The purpose of this article is to review the current status of gene therapy in light of recent successes and tragedies, and to consider the challenges faced by this relatively new field.
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