Gene therapy has cured children who have severe combined immunodeficiency (SCID), without so far causing cancer as previous treatment forms did.
David Williams at Boston Children's Hospital in Massachusetts, Alain Fischer of the Necker Hospital for Sick Children in Paris and their co-workers made a viral vector containing a corrected version of the mutated gene that otherwise hobbles the immune systems of children with SCID. Nine boys were treated; eight survived during the 1–3-year follow-up period, while one died of an infection that predated the treatment.
The researchers deleted certain key sections of the viral vector's DNA and found that the virus did not insert itself as often into cancer genes in the patients' genomes as earlier versions of the virus did. None of the boys has yet developed cancer, but the researchers note that only long-term monitoring will rule out that possibility.
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Another try at gene therapy for SCID. Nature 514, 277 (2014). https://doi.org/10.1038/514277a
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DOI: https://doi.org/10.1038/514277a
