Article ID | Journal | Published Year | Pages | File Type |
---|---|---|---|---|
6115343 | Current Opinion in Immunology | 2012 | 5 Pages |
Abstract
⺠Gene therapy provided a clinical benefit to most of the 60 patients affected by ADA-deficiency or SCID-X1. ⺠Gene therapy can be a valuable alternative to allogeneic hematopoietic stem cell transplantation. ⺠Insertional oncogenesis still represents the major issue to solve. ⺠Homologous recombination is rapidly improving to edit gene mutations.
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Authors
Marina Cavazzana-Calvo, Alain Fischer, Salima Hacein-Bey-Abina, Alessandro Aiuti,