Gene Therapy--The Next Generation

Vectors in development seek to benefit from earlier trials The gene therapy field resembles a toolbox containing instruments researchers haven't quite mastered, and the number of devices--viral and nonviral vectors--in this toolbox keeps increasing. "There are all these different tools out there," notes A. Dusty Miller, researcher at Fred Hutchinson Cancer Research Center in Seattle. "People are still trying to figure out what tools to use for what diseases." Photo: Richard Lobell Photography

Written byPaul Smaglik

| 9 min read

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Photo: Richard Lobell Photography

BIOBYPASS: During a normal open-heart surgery, a surgeon injects an adenovirus carrying a molecule promoting endothelial cell growth into parts of the heart where a normal bypass can't be effective. Insulin syringes (inset) contain the vectors.

Miller and other gene therapy investigators will report on the strengths and weaknesses of a host of vectors at the American Society of Gene Therapy's inaugural meeting in Seattle May 27-31. James M. Wilson, director of the Institute for Human Gene Therapy at the University of Pennsylvania Medical Center in Philadelphia, notes that all vectors have one goal in common: delivering therapeutic material into cells. However, most similarities end there:

Gene therapy researchers must find ways to improve each vector, as well as match vector characteristics with diseases they will most successfully target. Wilson emphasizes that there likely won't be one universal vector but rather a portfolio of specialized ones--some ...

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