Allied Academies

Gene Therapy For years we have known that many diseases have their origin at a cellular or genetic level, arising through single or multiple genetic errors. The advent of cell and gene therapy is giving us the potential to repair and replace cells and genes to cure rather than just control diseases. So, how are these therapies currently being applied in the 21st century? Gene therapy involves the introduction of a small amount of new genetic material into cells using a disarmed viral vector carrier. This genetic material can either be integrated into the chromosome of the cell or expressed within the cell depending upon the therapeutic approach. The use of this approach for disease treatment was first tried over 20 years ago and Cobra’s founding company Therexys was one of the first companies to be set up within the UK to assess this approach. However, it is only in recent years that researchers and clinicians have been able to achieve the clinical successes they first hoped fo...

For gene therapy  research, the perennial challenge has been devising a reliable way to insert safely a working copy of a gene  into relevant cells that can take over for a faulty one. But with the recent discovery of powerful gene editing  tools, the landscape of opportunity is starting to change. Instead of threading the needle through the cell membrane with a bulky gene, researchers are starting to design ways to apply these tools in the nucleus—to edit out the disease-causing error in a gene and allow it to work correctly. While the research is just getting underway, progress is already being made for a rare inherited immunodeficiency called chronic granulomatous disease (CGD). As published recently in Science Translational Medicine, a team of NIH researchers has shown with the help of the latest CRISPR/Cas9 gene-editing tools, they can correct a mutation in human blood-forming adult stem cells  that triggers a common form of CGD. What’s more, the...

CAR-T takes T-cells, cells which seek out and/or destroy disease in the body and uses synthetic DNA to alter them so they can recognize cancer cells via a specific protein (of the scientists choice) on the cancer cell surface. When put back into the patient the CAR-T cells can now seek out cancer cells and destroy them by signaling the bodies own immune system to attack cancer, much like it would an infection. This one of the most promising new cancer therapies available, with the first drug of its kind (Kymriah), approved last year. However, CAR-T is not without fault. The study highlights two major issues with CAR-T, using T-cells which seek out Folate receptor α (FRα), a protein on the surface of several cancers including ovarian3. First, they show that the CAR-T cells select for tumour cells which don’t have FRα, killing the ones with it and leaving a population without FRα behind; this essentially “evolves” a tumour to escape the treatment. Second, the tumour is often an...