Biomedical Engineering Reference
In-Depth Information
100
80
60
40
20
0
Figure 13.1
Variations in the number of human gene transfer protocols, 1988-2003.
The major conditions targeted in the “other diseases or disorders”
category
•
Peripheral artery disease: 20
•
Coronary artery disease: 19
By type of infectious disease studied
•
HIV infection or AIDS: 37
The Most Successful Gene Transfer Study to Date
Despite the extensive efforts of U.S. researchers in more than five
hundred disease-oriented gene transfer protocols, the clearest example
so far of success in a human gene transfer clinical protocol occurred in
France, where Doctor Alain Fischer and his colleagues at the Necker
Hospital in Paris treated several young male children who had inherited
X-linked severe combined immunodeficiency. In this disorder, neither of
the two major components of the immune system functions properly. In
Fischer's study, the children received their own genetically modified
bone marrow cells, presumably including bone marrow stem cells. The
modified cells seemed to have a competitive advantage over the native,
malfunctioning cells, and produced positive results in all but one child.
Fischer and his colleagues reported their positive results in
Science
in
April 2000, and the
New England Journal of Medicine
in April 2002.
2
Much to everyone's regret, news reports from early October 2002 and
additional information published in January 2003 indicated that two of
the first nine children given gene transfer in this protocol had developed
T cell leukemias. The retroviral vector used in Fischer's study activated
