Current status and prospects for gene therapy

• Published in: Vox sanguinis Vol. 87; no. 2; pp. 73 - 81
• Main Authors: Nathwani, A. C., Benjamin, R., Nienhuis, A. W., Davidoff, A. M.
• Format: Journal Article
• Language: English
• Published: Oxford, UK Blackwell Science Ltd 01-08-2004
• Subjects: AAV vectors; adenoviral vectors; Child; Clinical Trials as Topic; Forecasting; gene therapy; Gene Transfer Techniques; Genetic Therapy - adverse effects; Genetic Therapy - trends; Genetic Vectors; Hemophilia A - therapy; Humans; Neoplasms - therapy; oncoretroviral vectors
• ISSN: 0042-9007; 1423-0410
• DOI: 10.1111/j.1423-0410.2004.00543.x

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.