For acquired diseases, such as cancer, gene-therapy trials are introducing genes that are intended to kill cancerous cells.
ECONOMIST: The prospects for using genes as a therapy may be improving
Direct injection is also being used in gene-therapy trials on patients with Parkinson's and on those with muscular dystrophy.
ECONOMIST: The prospects for using genes as a therapy may be improving
Patient groups are sponsoring drug discovery at other firms, including Iceland's Decode Genetics and England's Oxford BioMedica, which is plying a gene-therapy approach.
Last month, the European Union approved Glybera for treatment of a rare genetic disease, making it the first gene-therapy medicine approved in the Western world.
At the moment, the discussion is about gene-therapy for embryos.
The idea suffered major setbacks in 1999 when a U.S. teenager died in a gene-therapy trial and again soon after when several children in Europe developed leukemia after receiving gene therapy.
In 2003 the company's treatment for head and neck cancer, which accounts for about 10% of the 2.5m new cancer patients in China every year, gained the first commercial approval of a gene-therapy treatment.
ECONOMIST: The prospects for using genes as a therapy may be improving
The work is significant because it goes a long way towards proving that the concept of gene-targeted therapy can work in major common tumors.
It is working on better protein-based drugs, gene therapy, industrial enzymes, and ag-biotech.
Most prominently, teenager Jesse Gelsinger died from multiple organ failure a few days after receiving a massive dose of virus-based gene therapy in a trial at the University of Pennsylvania.
But Dr. Gage also has a long-standing interest in gene therapy.
GlaxoSmithkline today unveiled a surprising deal to commercialize a new gene therapy technique for an ultra-rare type of immune deficiency.
Late in the summer ten kids with advanced neuroblastoma, one of the deadliest childhood cancers, began undergoing a radical new gene therapy with a breathtaking goal--to fight the disease not by deploying often inadequate chemotherapy and radiation but by jiggering the immune system to attack tumors it otherwise ignores.
Professor Nevin said that cancer was always seen as a possible side-effect of this type of gene therapy.
It will move the IL-12 drug and other cancer gene therapy approaches through human trials while Intrexon continues to work on basic science.
It turned out that the virus used to deliver the gene therapy was also inserting itself into cancer-causing areas of the genome, and not just where it was needed to cure the kids.
Such a treatment killed 18-year-old Jesse Gelsinger in 1999 in a gene therapy experiment.
The nastiest-looking arteries might be stabilized with some form of local therapy, such as drug-coated stents, light-activated drugs to kill off inflamed cells or even gene therapy.
The Glaxo gene therapy program will focus on a severe immune deficiency called ADA-SCID, in which kids cannot make a crucial enzyme called adenosine deaminase that is needed to create immune system cells.
Its technique involves extracting a patient's own bone-marrow cells, isolating certain stem cells, and delivering the gene therapy before returning the cells to the body.
McCarty, an investigator at Nationwide's Center for Gene Therapy, says relying on another researcher's data from a natural-history study would be problematic.
After years of controversy, gene therapy is poised to become a viable option for a variety of often life-threatening medical conditions, especially those resulting from a single defective gene.
Besides Genzyme General, the gene therapy program is involved in Genzyme Molecular Oncology gzmo (nasdaq: gzmo - news - people), which focuses on cancer vaccines, and Genzyme Surgical Products gzsp (nasdaq: gzsp - news - people).
The gene therapy experiment was largely funded by Project ALS, an innovative charity founded by ALS-sufferer Jenifer Estess and two of her sisters.
The Ziopharm-Intrexon deal gives Kirk an opportunity for a payday no matter what happens with the gene therapy.
Conscious that it needed a drug pipeline to survive as a drug company, Bayer has made deals with cutting-edge firms like genomics leaders Curagen and Millennium Pharmaceuticals , as well Avigen , a gene therapy company, and Lion Bioscience , a biotech computing company.
Shire research is aimed at developing an enzyme-replacement therapy, while the planned research at Nationwide Children's will focus on potential gene therapy.
Quake's technology "could allow biologists to do all sorts of things that we want to do that either aren't possible or are too time-consuming and expensive to do on a macroscopic scale, " says W. French Anderson , a gene therapy pioneer at the University of Southern California.
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