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- MEDICINE, Page 61Green Light
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- Scientists stand on the brink of performing gene therapy
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- The goal is grand -- and maddeningly difficult to achieve.
- Ever since Watson and Crick first deciphered the structure of
- DNA in 1953, doctors have had visions of treating disease not
- from the outside, with drugs or scalpels, but from the inside,
- by altering the primal instructions tucked in the nucleus of
- living cells.
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- Now, after years of debate about the ethics of genetic
- engineering and lengthy tests in animals, the first human
- trials are about to begin. Last week two experimental
- techniques passed a major regulatory hurdle, winning approval
- from the National Institutes of Health's Recombinant DNA
- Advisory Committee. The official go-ahead from the director of
- the NIH, as well as a nod from the Food and Drug Administration,
- is expected to follow within a few months for at least one of
- the experiments, clearing the way for human gene treatments as
- early as this fall. "This is the first step toward what will
- probably be a medical revolution," said Dr. W. French Anderson,
- one of the scientists whose proposal was approved. "Millions
- of patients are going to be helped by this in the future."
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- The two experiments rely on a technology that has evolved
- over the past 12 years. Each uses a virus to act as a kind of
- biological taxi to transport a desired gene into the nucleus
- of human blood cells. In one experiment, a team led by Dr.
- Steven Rosenberg proposes to treat malignant melanoma, a form
- of skin cancer, with blood cells that have been genetically
- altered to transform them into tiny factories for a
- tumor-killing protein.
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- The experiment proposed by Dr. Anderson is more
- controversial. He would use gene therapy to treat children who
- lack a key immune-system enzyme called adenosine deaminase
- (ADA), leaving them vulnerable to every passing germ. Some
- researchers question the wisdom of performing a novel -- and
- potentially dangerous -- therapy on children, especially since
- there is already an effective drug treatment. "There are a lot
- of other diseases without therapies," says Duke University's
- Dr. Michael Hershfield, an expert on ADA deficiency. "And
- they're in adults who can make decisions for themselves."
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- Thousands of inherited diseases may be linked to the
- malfunctioning of specific genes. In addition, researchers are
- discovering that nearly every disorder has a genetic component.
- Last week the National Cancer Institute published two studies
- suggesting that susceptibility to lung cancer may be associated
- with a single gene.
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- But there is no guarantee that gene therapy will be
- effective against any of these illnesses. Some genes are too
- big to fit inside the viral taxi. And things could go wrong.
- The new genes might not "turn on" inside the body, or they
- might get misplaced in the gene sequence and rather than fight
- cancers, start triggering them instead. Ultimately, the only
- way to see what happens is with carefully designed experiments.
- As Dr. Anderson puts it, "Now we can find out if gene therapy
- is really going to work."
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- By Philip Elmer-DeWitt. Reported by Dick Thompson/Washington.
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