About genetic diseases

Build an understanding of genetics

Before we can jump into gene therapy, we need to start with genetics. This will help you understand how your DNA makes you the unique person that you are.

Commonly used terms in genetics

Deep inside every part of your body lies special information called genetic information. It is passed down to you by your birth parents and helps shape what you look like and how your body works.

Cells: The smallest unit of life. Your body is made up of trillions of cells.

Chromosomes: Within each cell there are 23 pairs of chromosomes that you inherit from your birth parents.

DNA: Each chromosome is made of strands of deoxyribonucleic acid, also known as DNA. DNA carries the genetic information needed to build and maintain everything in your body.

Genes: Your DNA is made up of different sections called genes. Each gene is responsible for creating proteins that perform different tasks.

Proteins: Proteins are created by genes to carry out specific tasks in your body. Some of these tasks shape how you look or your body’s ability to work smoothly.

Cell with magnification of a chromosome inside of the cell
Cell with magnification of a chromosome inside of the cell

Why is genetics important?

You can think of your genes as an instruction manual within your cells, giving your body directions for what to do and how to do it. Genes provide instructions for physical qualities such as:

They also give instructions to make your body work:

When the instructions are correct, your body runs smoothly.
When they are incorrect, your body may not function as intended, which could lead to a genetic disease.

Learn what causes genetic diseases
Instruction manual icon with finger pointing to  represent how genes are like an instruction  manual

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