Going viral: How the first FDA approved gene therapy to treat an inherited disease uses a virus to deliver a functional gene and what the large price tag may mean for patients.
“When you think of viruses, the yearly flu or even the Ebola or Swine flu outbreaks may come to mind. However, not all viruses cause disease – some even provide cures!
Adeno-associated virus (AAV) can infect humans, but is not known to cause disease. In other words, this virus is good at getting its genetic information (genes) into human cells. What if its genes were replaced with human genes related to disease? Could we cure genetic disease by giving cells a healthy copy of the mutated gene? These questions helped spark the field of gene therapy. …”
via Viruses, not all are bad for you — Science in the News
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