Therapy Researchers

Sidebar: Summary Table of Vectors

PROGRESS: Forrest Anthony says some FDA concerns have been allayed.
Despite safety questions from federal agencies, the gene therapy industry is hotly pursuing new vectors. Since 1990, dozens of biotech and gene therapy companies have hired life scientists and launched research programs to find safe, effective carriers to shuttle therapeutic genes into the body.

According to the Pasteur Institute, a nonprofit research organization in Paris, 216 gene therapy protocols with human patients-treating diseases such as cystic fibrosis, cancer, and AIDS-are under way worldwide.

In the vast majority, therapeutic genes are packed into disarmed viruses, which shuttle the genetic payload into a cell's chromosomes.

These viral vectors require the expertise of virologists, biochemists, and molecular biologists, among others. Meanwhile, nonviral vectors-for example, synthetic molecules that tote therapeutic genes-call for biophysicists and analytical chemists.

Gene therapy researchers agree that finding-or creating-viable vectors is of...

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