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Gene therapy - promises, problems and prospects

Nature volume 389, pages 239–242 (1997)Cite this article

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In principle, gene therapy is simple: putting corrective genetic material into cells alleviates the symptoms of disease. In practice, considerable obstacles have emerged. But, thanks to better delivery systems, there is hope that the technique will succeed.

Box 1 In 1990, the first clinical trials for gene-therapy approaches to combat disease were carried out. Conceptually, the technique involves identifying appropriate DNA sequences and cell types, then developing suitable ways in which to get enough of the DNA into these cells. With efficient delivery, the therapeutic prospects range from tackling genetic diseases and slowing the progression of tumours, to fighting viral infections and stopping neurodegenerative diseases. But the problems — such as the lack of efficient delivery systems, lack of sustained expression, and host immune reactions — remain formidable challenges.

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Figure 1: To create the retroviral vectors that are used in gene therapy, the life-cycles of their naturally occurring counterparts are exploite.

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Authors and Affiliations

  1. the Laboratory of Genetics, The Salk Institute for Biological Studies, La Jolla, 92037, California, USA

    Inder M. Verma & Nikunj Somia

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  1. Inder M. Verma
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Verma, I., Somia, N. Gene therapy - promises, problems and prospects. Nature 389, 239–242 (1997). https://doi.org/10.1038/38410

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