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Science 26 October 1984: Vol. 226. no. 4673, pp. 401 - 409 DOI: 10.1126/science.6093246
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Articles
Science, Vol 226, Issue 4673, 401-409
Copyright © 1984 by American Association for the Advancement of Science
Prospects for human gene therapy
WF Anderson
Procedures have now been developed for inserting functional genes into the bone marrow of mice. The most effective delivery system at present uses retroviral-based vectors to transfer a gene into murine bone marrow cells in culture. The genetically altered bone marrow is then implanted into recipient animals. These somatic cell gene therapy techniques are becoming increasingly efficient. Their future application in humans should result in at least partial correction of a number of genetic disorders. However, the safety of the procedures must still be established by further animal studies before human clinical trials would be ethical.
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