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    Gene Therapy: The Future Is Now


    The disease involved in the French research, and in Anderson's early work, is called severe combined immunodeficiency disease (SCID), and is very rare. Genetically speaking, it affects a single gene, so it is more basic than many other diseases.

    But the proof is now in the pudding, and there's more to come. Already, advances in cancer, heart disease, and others are close enough to taste.

    The new research "establishes as a fact that if you can get enough gene-corrected cells into the body, you can correct the disease," Anderson tells WebMD. "So it's been done first with SCID; now it's a matter of working out the conditions for other diseases. In fact, hemophilia [treatment], looks like that's going to be successful; growing new blood vessels for cardiovascular disease looks successful; some of these gene-based vaccines for cancer ... those look very promising." Using gene therapy to treat the AIDS virus also shows huge potential, he says.

    It will take longer to find solutions to certain diseases like diabetes and sickle cell anemia, Anderson says, because they involve multiple genes. "We're just getting over the cusp of being able to get a single gene in, much less being able to get two genes in or three genes, and have them [work] properly, and so on," he tells WebMD.

    Some researchers are also having success with gene therapy in mice. "There are a number of indications now that finally we've learned enough; we've made the system efficient enough that gene therapy is now turning the corner and is starting to see some success clinically," Anderson says.

    Fears about safety have also calmed down, according to Anderson. He says that out of hundreds of recent problems associated with clinical trials that were investigated by the government, only one was linked to a gene-related event: the death of the Arizona teen-ager, Jesse Gelsinger.

    Is humanity facing a cure-all approach to treatment? Anderson tells WebMD researchers are working on it. "Not quite that completely, but the answer is yes -- but we're talking about 20, 30, 40 years. It is recognized that gene therapy will revolutionize medicine. But the problem is, we all got a bit carried away into thinking it was going to happen easily. Well, nothing major happens easily in nature.

    "My feeling is for the next three to five years, all we're going to see is the occasional, little indication. I think we've got another five years of hard work before gene therapy really starts to become mainline."

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