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Current status and prospects for gene therapy

Nathwani, AC; Benjamin, R; Nienhuis, AW; Davidoff, AM; (2004) Current status and prospects for gene therapy. VOX SANG , 87 (2) 73 - 81.

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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.

Type: Article
Title: Current status and prospects for gene therapy
Keywords: gene therapy, oncoretroviral vectors, adenoviral vectors, AAV vectors, SEVERE COMBINED IMMUNODEFICIENCY, HEMATOPOIETIC STEM-CELLS, HIGH-LEVEL EXPRESSION, ADENOASSOCIATED VIRUS, FACTOR-IX, ADENOVIRAL VECTORS, IMMUNE-RESPONSES, HEMOPHILIA-B, FACTOR-VIII, DELIVERY
UCL classification: UCL > Provost and Vice Provost Offices
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Cancer Institute
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Cancer Institute > Research Department of Haematology
URI: http://discovery.ucl.ac.uk/id/eprint/1330933
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