Gene Therapy Review
Welcome to our gene therapy portal
The Gene Therapy Review is the web resource for patients and professionals interested in cell and gene therapy. Founded in 2008, the aim of the site is to be an information resource for basic and clinical research in the fields of gene therapy, cell therapy, and genetic vaccines, and to serve as a network in the exchange of information and news related to this arena. We welcome contributions from researchers and clinicians alike with a view in increase awareness in this vastly growing sector.
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.