What is Cell & Gene Therapy?
Cell and gene therapies aim to introduce reparative (repairing) cells to correct the effect of a disease-causing gene – and thereby treat the underlying cause of the medical problem.
Gene therapy is the insertion of genes into an individual’s cells and tissues to treat disease, in particular, hereditary diseases.
Gene therapy typically aims to supplement a defective gene with a functional one – a normal gene is inserted into the genome, to replace the function of an abnormal disease-causing gene.
The gene therapy is delivered using a special DNA-containing ‘vector’ – the most common type of vectors are viruses, genetically modified to carry normal human DNA – Mother Nature’s own nanotechnology!
Viruses have evolved to deliver their own genes to human cells – so we have tried to harness this ability by removing disease-causing genes and insert therapeutic ones.
Diseased cells such as the patient’s liver or lung cells are targeted by the vector. The vector then unloads the therapeutic human gene into the target cell.
The generation of a functional protein product from the therapeutic gene restores the target cell to a normal state.