Gene Therapy Conference, Uncategorized

Increasing Opportunities in Cell & Gene Therapy

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? Continue reading “Increasing Opportunities in Cell & Gene Therapy”

Gene Therapy Conference

Find and Replace: DNA Editing Tool Shows Gene Therapy Promise

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. Continue reading “Find and Replace: DNA Editing Tool Shows Gene Therapy Promise”

Gene Therapy Conference

Viruses, BiTEs, and T-cells: A Triple Threat to Tackle Cancer:

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. Continue reading “Viruses, BiTEs, and T-cells: A Triple Threat to Tackle Cancer:”