gene-cell-therapy

Gene Therapy is the transfer of genetic material (gene transfer) to dysfunctional cells to correct a deficiency in the DNA or genome of a patient. Cell Therapy, such as stem cell therapy, can also be used to achieve these goals. These approaches can be applied to genetic disorders as well as diseases acquired over the lifetime of an individual, such as cancer or infection, to confer a specific property to the cell allowing it to combat the disease.

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Gene Therapy in the News

  • Mutation of one gene is all it takes to get cystic fibrosis, but disease severity depends on many other genes and proteins. For the first time, researchers identified genetic pathways that play major roles in why one person with CF might have severe symptoms while another person might not.

  • A novel gene-delivery system that shuttles a gene into a cell, but only for a temporary stay, has been developed by researchers, providing a potential new gene-therapy strategy for treating disease. The approach offers distinct advantages over other types of gene therapies currently under investigation, said the lead author of a study describing the new technique.

  • Nanoparticles have been used to successfully deliver a new therapy to cancer cells in the brains of rats, prolonging their lives, scientists report. Previous research on mice found that nanoparticles carrying genes can be taken up by brain cancer cells, and the genes can then be turned on. However, this is the first time these biodegradable nanoparticles have effectively killed brain cancer cells and extended survival in animals.

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© 2012 Gene Therapy Review. All Rights Reserved. ISSN (print) 1792-0094, ISSN (online) 1792-0108