The Long Horizon of Gene Therapy
A. The human body has much better defenses against manipulating its DNA than we thought it had. We can do miracles in a tissue culture dish. You can cure cancer in mice with gene therapy. But then you go to the human body, and we face three major problems.
The first is, how do you get a gene into enough cells to make a difference? The second is the body's defense mechanisms, which basically shut down the genes once they get there. And then the third is figuring out how to get genes into cells at the right place, at the right time, making the right amount of product to treat a given disease......
Q. Look into your crystal ball. Is the business outlook for gene therapy still exciting?
A. All around the world, there is progress being made. The first indications that we might be tipping over the hump are in cardiovascular disease, cancer, AIDS and hemophilia. We are seeing signs that there are potential products, real products.
Over the next two or three years, if we don't hit another brick wall, and several of these products get into Phase 3 trials and things look encouraging, the business outlook for gene therapy will brighten again and money will flow back into the field.
copywrite NY Times December 12, 1999
nytimes.com
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