Policy ForumMedicine

Paying for future success in gene therapy

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Science  27 May 2016:
Vol. 352, Issue 6289, pp. 1059-1061
DOI: 10.1126/science.aaf4770

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Summary

Imagine a young man with hemophilia A who no longer has to self-administer factor VIII replacement; an individual with sickle cell disease who is free of chronic pain and intermittent crises; a girl functionally blind since the age of 5 who can now see; or a baby rescued from a fatal, inherited neurodegenerative disease. For decades, gene therapy has tantalized us with such futuristic scenarios. However, these goals are now coming into focus, and it is the time to consider some of the consequences of success.