Resources

Glossary

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

 

CRISPR (KRIS-per)

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

 

DNA

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

 

IV

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

 

RNA

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

 

ZFNs

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”