Gene replacement therapy is being studied as a treatment option for genetic diseases, and we want to help you understand the science behind it.
To begin, let’s talk about genes. Genes are small sections of DNA. DNA is inherited from your parents and carries instructions that tell the body how to function properly. Specific genes tell the body how to make specific proteins. Proteins play important roles in the body, like helping your cells to function properly or acting as the building blocks of your body. If a gene has an error, and the body can’t make a specific protein, it can be damaging to a person’s health.
A genetic disease or disorder is the result of an error in one or more of a person’s genes. The disease a person has depends on which gene in their DNA has the error. Depending on the disease or disorder, it can be inherited from one or both parents. Or, sometimes it’s a change that just happens randomly. A genetic disease caused by a single gene that is faulty or missing is called a monogenic disease.
Watch the video explainer or read the full transcript on AveXis website.