Recent clinical trials of gene therapy have shown remarkable therapeutic benefits and an excellent safety record. They provide evidence for the long-sought promise of gene therapy to deliver 'cures' for some otherwise terminal or severely disabling conditions. Behind these advances lie improved vector designs that enable the safe delivery of therapeutic genes to specific cells. Technologies for editing genes and correcting inherited mutations, the engagement of stem cells to regenerate tissues and the effective exploitation of powerful immune responses to fight cancer are also contributing to the revitalization of gene therapy.
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L.N. apologizes to the many scientists whose contributions to the field could not be acknowledged owing to space limitations. He thanks past and present members of his laboratory and colleagues at the San Raffaele Telethon Institute for Gene Therapy (TIGET) and the San Raffaele Scientific Institute. L.N. is also grateful to the Telethon Foundation, the European Union (FP7 and ERC), the Italian Association for Cancer Research, and the Italian ministries of health and of scientific research for supporting his research.
L.N. is listed as an inventor on pending and issued patents on lentiviral-vector technology, microRNA-regulated vectors and targeted genome editing that were filed by the Salk Institute for Biological Studies, Cell Genesys, the Telethon Foundation and the San Raffaele Scientific Institute. As the director of TIGET, L.N. is involved in a strategic alliance for the development (to marketing authorization) of HSC-based gene therapies for some rare diseases with GlaxoSmithKline (GSK), which licensed metachromatic leukodystrophy and WAS gene therapies in 2014 and became the financial sponsor of the trials. L.N. has established research collaborations on targeted genome editing in HSCs with Sangamo BioSciences and on lentiviral gene therapy of haemophilia with Biogen. L.N. is a founder, owns equity in and chairs the scientific advisory board of Genenta Science, a biotechnology start-up company that aims to develop gene therapy for tumours using tumour-infiltrating monocytes.
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Naldini, L. Gene therapy returns to centre stage. Nature 526, 351–360 (2015). https://doi.org/10.1038/nature15818
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