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Department of Microbiology & Immunology University of Illinois Chicago The gene defective in cystic fibrosis patients was identified and sequenced last year. Now that the quite different research skills of physiology and molecular genetics groups have been put together, the wild-type normal gene has been transfected into cultured defective cells and shown to work. There still is a long, long way to go before the disease can be cured by gene therapy. Already the cloned gene will be useful in d

Simon Silver
Nov 11, 1990

Department of Microbiology & Immunology
University of Illinois Chicago

The gene defective in cystic fibrosis patients was identified and sequenced last year. Now that the quite different research skills of physiology and molecular genetics groups have been put together, the wild-type normal gene has been transfected into cultured defective cells and shown to work. There still is a long, long way to go before the disease can be cured by gene therapy. Already the cloned gene will be useful in diagnosis and in developing drug therapy. Developing methods for actual gene replacement in dysfunctional cells in humans will be much more difficult.

M.L. Drumm, H.A. Pope, W.H. Cliff, J.M. Rommens, et al., "Correction of the cystic fibrosis defect in vitro by retrovirus-mediated gene transfer," Cell, 62, 1227-33, 21 September 1990. (University of Michigan, Ann Arbor; University of Alabama, Birmingham; Hospital For Sick Children, Toronto)

D.P. Rich, M.P. Anderson, R.J. Gregory,...

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