Glossary of terms

Deletion is a form of genetic change, or mutation, where a piece of the DNA sequence is missing or removed from the genome, resulting in the loss of genetic information at that location. Deletion of a gene, or a part of it, can change the way the body develops and functions, and be a cause for genetic disease. However, deletions in the genome can also occur without any harmful impact and may even be beneficial to the organism.

'Delivery' refers to the method by which a therapeutic agent (such as a drug or modified cells) enters the body and travels to the correct location to be activated. DIfferent therapies require different delivery methods to be effective. These can range from an infusion to an oral pill to an implanted device.

Designer nucleases are engineered enzymes designed to target and modify specific DNA sequences in the genome with high precision. They are used in genome editing technologies such as CRISPR-Cas9, zinc finger nucleases (ZFNs), and transcription activator-like effector nucleases (TALENs). These nucleases can induce DNA breaks at desired locations, allowing for precise modifications such as gene knockout, gene insertion, or gene correction.

DNA (Deoxyribonucleic acid) is a molecule composed of the four building blocks (bases) adenine (A), cytosine (C), guanine (G) and thymine (T) structured into two chains that coil around each other to form a double helix carrying genetic instructions for the development, functioning, growth, and reproduction of all known organisms and many viruses. This genetic code is the blueprint for the production of proteins and other functional molecules that are essential for the life and health of the organism. DNA is found in the nucleus of cells, is organized into chromosomes, and is responsible for storing and transmitting genetic information from one generation to the next.