Abstract
Over 70 human diseases have been named as potential beneficiaries of somatic cell gene therapy, ranging from inherited disorders, such as cystic fibrosis and forms of severe combined immune deficiency, to cancer and AIDS (Scarpa and Caskey, 1989). Intervention by ‘designer genes’ incorporated into patients’ own cells could replace absent or defective molecules, synthesize metabolic enzymes or drugs, or manipulate the body’s immune responses (Ledley, 1990; Miller, 1990a). The realisation that recombinant DNA technology, now familiar in a diagnostic context, can be applied to the correction of diagnosed diseases is leading to a renewed optimism in the clinic for treating conditions that have so far persistently defeated more conventional therapeutic strategies.
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Dunckley, M.G., Dickson, G. (1994). Retroviral-Mediated Gene Transfer and Duchenne Muscular Dystrophy. In: Wolff, J.A. (eds) Gene Therapeutics. Birkhäuser Boston. https://doi.org/10.1007/978-1-4684-6822-9_22
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DOI: https://doi.org/10.1007/978-1-4684-6822-9_22
Publisher Name: Birkhäuser Boston
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