Clinical Research: New Zealand's Leap Into Gene Therapy

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Science  15 Mar 1996:
Vol. 271, Issue 5255, pp. 1489-1490
DOI: 10.1126/science.271.5255.1489


New Zealand's first-ever gene therapy experiment took place last week when researchers at Auckland Medical School treated two children with Canavan disease, the disease that figured in the movie Lorenzo's Oil. The test was the first targeted at a neurological disease other than brain cancer, and it used a novel “vector” to insert genes into the target cells. The chances of therapeutic benefit are considered slim, however. The treatment was developed mostly in the United States, where clinical trials of gene therapies must be approved by the Recombinant DNA Advisory Committee. Some RAC members have raised concerns that it may set a precedent for moving clinical tests outside RAC's jurisdiction.