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Viral vectors carry the risks of toxicity, inflammatory responses, and gene control and targeting issues.
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Some commonly occurring disorders, such as heart disease, high blood pressure, Alzheimer's disease, arthritis, and diabetes, are affected by variations in multiple genes, which complicate gene therapy.
Some therapies may breach the Weismann barrier (between soma and germ-line) protecting the testes, potentially modifying the germline, falling afoul of regulations in countries that prohibit the latter practice.
Alipogene tiparvovec or Glybera, e.g., at a cost of $1.6 million per patient, was reported in 2016 to be the world's most expensive drug.
Today, we know that the "transforming principle" Griffith observed was the DNA of the III-S strain bacteria. While DNA had been known to biologists since 1969, most assumed at the time that proteins carried the information for inheritance.
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