A team of scientists led by Dr. Luigi Naldini at the San Rafaele Telethon Institute for Gene Therapy (HSR-TIGET) in Milan have developed a new platform for regulating the expression of therapeutic genes, which will have major implications for gene and cell therapy. Dr. Naldini also presented his team's findings on therapeutic use of human stem cells, including potential application to X-linked Severe Combined Immunodeficiency (XSCID or Bubble-Boy disease). Dr. Anthony Cheung, from where, presented a safer method to deliver the insulin gene to juvenile diabetes patients using nanoparticles, developed with his colleagues at enGene, Inc., Vancouver. Finally, Dr. Krystof Bankiewicz, University of California-San Francisco, have developed an improved viral vector delivery system that allows real-time, direct MRI monitoring of the distribution of a gene transfer vector within the brain.
The American Society of Gene Therapy is a professional non-profit medical and scientific organization dedicated to the understanding, development and application of gene and related cell and nucleic acid therapies and the promotion of professional and public education in the field. For more information, visit
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In this study researchers analyzed more than 150 drugs aimed at reducing the toxicity of the 5-FU against the benign cells, thus avoiding the reproduction of new carcinomas or other side effects. "We discarded compounds until we finally found six which have shown to be better than currently used drugs," says Caba P'rez.
Research on cell lines
So far, the study has been conducted on cell lines, and not on patients, using a new technique called Microarrays or DNA Chips, which enables the identification of the effects produced by drugs on each gene the lowest and most specific level that Medicine can deal with.
Our research," says Caba P'rez, has been conducted with absolute precision with the aim of obtaining the lowest possible concentration of drug producing the most significant effect on cancerous cells. Therefore, this study has shown the importance of the presence of some toxic compounds like chlorine, flourine or uracil in drugs. This presence is one of the variables used to develop new antitumor drugs.
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