This site is an educational service brought to you by BioMarin and is intended for US residents only. Many gene therapies for hemophilia A and B are being studied in people to determine if they are safe and effective. No gene therapies for hemophilia have been approved for use or determined to be safe or effective.

What is a gene?

You’ve probably heard about genes and how you got your hair color from one biological parent and your eye color from the other. But there is so much more to genetics and how genes work in the body.

GENES ARE SEGMENTS OF DNA

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The key role of genes is to provide the instructions for making proteins. Proteins are the building blocks of the body and serve important functions like tissue repair and helping blood to clot.

GENES ARE SEGMENTS OF DNA

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Think of DNA as the language used in your genetic instructions. DNA is made up of components called nucleotide bases that are like the letters of a word. You must have the correct nucleotide bases in the correct order for the gene to fulfill its intended purpose—producing proteins with normal function.

DNA stands for deoxyribonucleic acid. The 4 nucleotide bases responsible for gene construction are adenine (A), guanine (G), cytosine (C), and thymine (T). These nucleotides pair up with each other, A with T and C with G.

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A chromosome

They can be found in our chromosomes, which contain tens of thousands of known genes. Your chromosomes lie deep within a structure called the nucleus, which acts as the command center of the cells that make up your body.

Human cells typically contain 23 pairs of chromosomes. In males and females, 22 of those pairs look the same. The 23rd pair, also called the sex chromosomes, differs between males and females. Females have two copies of the X chromosome while males have a single pair of X and Y chromosomes.

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