GENE & CELL THERAPY 101

Gene Therapy basics

Gene therapy has been studied for more than 40 years and can help stop or slow the effects of disease on the most basic level of the human body - our genes. And to understand how it works, we’ll start at the basics.

CELL THERAPY BASICS

The human body contains over 200 different specialised cell types, such as muscle, bone or brain cells. These cells carry out specific functions within the body, necessary for the health of an organism. Injury, disease or ageing can lead to the loss of specialised cells from the body. In many cases, such loss is irreversible, meaning that the diseased or lost cells can no longer be replenished by healthy ones.

VECTORS 101

Vectors are essentially vehicles designed to deliver therapeutic genetic material, such as a working gene, directly into a cell.

DIFFERENT APPROACHES

Gene therapy, cell therapy, and gene editing are fields of biomedical research with a similar goal in mind: To treat disease by changing our bodies at a microscopic level.