Glossary

A type of gene therapy A technique that uses a gene or gene(s) to prevent, treat or cure a disease or disorder. Most gene therapies work by adding a healthy version of a gene to replace one that is defective or missing into the genome of particular cells. Some of these therapies use viral vectors to deliver genes into target cells. gene therapy A technique that uses a gene or gene(s) to prevent, treat or cure a disease or disorder. Most gene therapies work by adding a healthy version of a gene to replace one that is defective or missing into the genome of particular cells. Some of these therapies use viral vectors to deliver genes into target cells. that works by delivering genetic material that can directly edit pieces of DNA Short for deoxyribonucleic acid, DNA is a double-stranded molecule that serves as the genetic blueprint for living organisms. Composed of four chemical bases, DNA encodes the instructions necessary for protein synthesis and governs the development, function, and inheritance of traits in an organism. DNA Short for deoxyribonucleic acid, DNA is a double-stranded molecule that serves as the genetic blueprint for living organisms. Composed of four chemical bases, DNA encodes the instructions necessary for protein synthesis and governs the development, function, and inheritance of traits in an organism. within a cell. This changes the instructions the DNA encodes for, which ultimately results in an increase or decrease in the production of a certain protein and the restoration of proper cell function.