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

  • Researchers have reconstructed an ancient virus that is highly effective at delivering gene therapies to the liver, muscle, and retina. This discovery could potentially be used to design gene therapies that are not only safer and more potent than therapies currently available, but may also help a greater number of patients.

  • New methods for modifying the genome are currently widely discussed: Using CRISPR/Cas for instance, scientists can remove parts of the genetic code of a gene, thereby knocking it out. Furthermore, there are ways to inhibit translation of a gene into a protein. Both methods have in common that they impede production of a protein and should therefore have comparable consequences for an organism. However, it has been shown that consequences can differ, after a gene is either knocked, out or only blocked. Scientist now find that additional genes compensate for a knocked out gene and either attenuate consequences or completely...

  • The term intellectual disability covers a large number of clinical entities, some with known cause and others of uncertain origin. For example Down syndrome is due to an extra copy of chromosome 21 and Rett syndrome is in part caused by a mutation in the control switch gene called MeCP2. In other cases the mechanisms by which they are produced are not clearly identified. Now a research team has discovered a mechanism that identifies a cause of intellectual disabilities in these puzzling cases.

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