Cas enzyme (kass EN-zyme)

Cuts DNA at a precise location to allow gene editing to take place


Cell (sel)

Smallest unit of life that makes up all living things


Chromosomes (KRO-mo-zomes)

Folded strands of DNA that contain genes


Clinical trials (KLI-ni-kuhl TRI-ulz)

Studies designed to test the safety, efficacy, and effectiveness of a new therapy



Also known as clustered regularly interspaced short palindromic repeats. A type of gene editing that can change DNA in order to treat diseases



Also known as deoxyribonucleic acid (dee-OX-ee-RY-bo-noo-CLAY-ic). A part of a cell that carries genetic instructions for how the body works


Enzyme (EN-zyme)

A substance that causes chemical reactions inside the body


Ex vivo (ex VEE-vo)

A term used to describe a process that occurs outside of the body


Gene (jeen)

Sequence of DNA that tells the body how to make proteins


Gene addition (jeen uh-DI-shun)

Addition of a normal gene to treat a disease caused by a faulty gene


Gene editing (jeen E-dit-ing)

Process of changing the body’s genetic material at precise locations in the DNA to manage or treat a disease


Gene therapy (jeen THAIR-a-pee)

Treatment that changes cells to manage or treat a disease


Genetic disease (je-NE-tik di-ZEEZ)

An inherited disease caused by a harmful change in a person’s DNA


Genetics (je-NE-tiks)

The study of genes


Guide RNA

Tells the Cas enzyme the precise location to cut the DNA


In vivo (in VEE-vo)

A term used to describe a process that occurs inside of the body


Informed consent (in-FORMD kun-SENT)

Agreeing to undergo a procedure after the risks and benefits have been thoroughly explained; may also be referred to as treatment consent



Also known as intravenous (IN-tra-VEE-nuss). Giving fluids, medications, and other treatments directly into a vein


Mutation (myoo-TAY-shun)

Change in DNA that might have a harmful effect on the body


Patient advocate (pay-SHUNT AD-vo-cut)

A person who works for a hospital or healthcare facility that helps patients navigate their treatments, provides resources, and helps them find answers to their questions


Protein (PRO-teen)

Proteins give cells their shape and help the body work the way it should



Also known as ribonucleic (RY-bo-noo-CLAY-ic) acid. A part of a cell that converts the information in DNA to help make proteins


TALENs (TAL-enz)

Also known as transcription activator-like effector nucleases. A type of gene editing that works by combining 2 different types of proteins



Also known as zinc-finger nucleases (NOO-clee-AY-siz). A type of gene editing that’s made up of chains of proteins called “zinc fingers” and tiny DNA repair tools found in bacteria, known as “bacterial nucleases”