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Cell preselection is key to gene therapy success

BMJ 2000; 320 doi: https://doi.org/10.1136/bmj.320.7245.1296 (Published 13 May 2000) Cite this as: BMJ 2000;320:1296
  1. Abi Berger
  1. BMJ

    New cell selection strategies look likely to improve the success of gene therapy for blood disorders. Using a green fluorescent labelling technique, scientists at the Massachusetts Institute of Technology in Cambridge have discovered a trick that greatly improves the proportion of stem cells that successfully integrate the desired gene.

    Dr Philippe Leboulche and his team seem to have got around the common problem of “silencing,” whereby a new gene is successfully introduced into the genome of a stem cell but fails to be expressed once cell differentiation occurs.

    Although their work …

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