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

  • For the first time gene therapy for cystic fibrosis has shown a significant benefit in lung function compared with placebo, in a phase 2 randomized trial. The technique replaces the defective gene response for cystic fibrosis by using inhaled molecules of DNA to deliver a normal working copy of the gene to lung cells.

  • Researchers have developed an innovative, experimental gene transfer-based treatment for children with giant axonal neuropathy (GAN). This extremely rare genetic disorder causes children to gradually lose the ability to balance themselves, move their muscles and to feel certain sensations. Most children born with GAN do not survive beyond their early 20s because of progressive impairment of their ability to breath.

  • The development of targeted therapies has significantly improved the survival of melanoma patients over the last decade; however, patients often relapse because many therapies do not kill all of the tumor cells, and the remaining cells adapt to treatment and become resistant. Researchers have discovered a novel mechanism that can lead melanoma cells to develop resistance to drugs that target the protein BRAF.

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