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What has happened to gene therapy?

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Abstract

Trials have demonstrated the feasibility of gene therapy in correcting the gene defect in monogenic disorders such as severe combined immune deficiency and cystic fibrosis, but there are still obstacles and ethical issues to overcome.

Conclusion With appropriate research, better delivery strategies and adherence to good standard clinical research and practice, gene therapy research will lead to clinical implementation in monogenic and multifactorial disorders including cancer, neurodegenerative disorders and vascular disease.

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Authors and Affiliations

  1. Department of Medical Genetics, Belfast City Hospital, Lisburn Road, Belfast BT9 7AB, Northern Ireland, UK e-mail: nc.nevin@bch.n-i.nhs.uk Tel: +44-28-90263873; Fax: +44-28-90236911, , , , , , IE

    Norman C. Nevin

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  1. Norman C. Nevin
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Nevin, N. What has happened to gene therapy?. Eur J Pediatr 159 (Suppl 3), S240–S242 (2000). https://doi.org/10.1007/PL00014411

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  • DOI: https://doi.org/10.1007/PL00014411

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