Gene therapy revisited

In spite of problems and drawbacks, gene therapy moves forward

Since the death of Jesse Gelsinger 2 years ago during clinical trials at the University of Pennsylvania, gene therapy has maintained a low profile and receded from the public eye. But away from the headlines, researchers are, in fact, quietly making progress and are confident that, within the next decade, gene transfer will be elevated from its current experimental status to a therapeutic modality.

At the recent Emerging Technologies in Gene/Drug Therapy and Molecular Biology meeting, sponsored by the Regulon

Away from the headlines, researchers are quietly making progress with gene transfer

company (Mountain View) and the International Society of Gene Therapy and Molecular Biology, a group of about 100 scientists gathered in Corfu, Greece, to discuss advances in bringing gene therapy to the clinic. They still face major challenges: targeting the right gene to the right location in the right cells and expressing it at the right time, all while minimising any adverse reactions. But the scientists presented data on the development of viral and non-viral gene vectors, tissue- and disease-specific gene delivery and cell cycle control that indicate that the clinical use of gene transfer is becoming a tangible possibility.

http://www.nature.com/embor/journal/v2/n12/full/embor261.html