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It involves laboratory experiments in animals and tissue samples to further determine and evaluate the safety and of course the effectiveness of the new technique. Using lab animals however, poses an exclusive problem; there are limited animals that have single gene defects that cause problems in humans and there are none that have the human disease of enzyme deficiency. So in short the resources for experimentation are very limited, time consuming and have to be induced to the animal by inserting a new gene into the animal cell and monitor the gene and even by doing this, it still will not be able to demonstrate a clinical "cure" in a diseased laboratory animal...
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