Transfer of Genes to Humans: Early Lessons and Obstacles to Success

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Science  20 Oct 1995:
Vol. 270, Issue 5235, pp. 404-410
DOI: 10.1126/science.270.5235.404


Enough information has been gained from clinical trials to allow the conclusion that human gene transfer is feasible, can evoke biologic responses that are relevant to human disease, and can provide important insights into human biology. Adverse events have been uncommon and have been related to the gene delivery strategies, not to the genetic material being transferred. Human gene transfer still faces significant hurdles before it becomes an established therapeutic strategy. However, its accomplishments to date are impressive, and the logic of the potential usefulness of this clinical paradigm continues to be compelling.

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