Genetic Therapies What Are Genetic Therapies?

Genetic therapies aim to treat or cure conditions by correcting problems in your
DNA

DNA

(NULL):

:The molecules inside cells that carry information and pass it from one generation to the next.

. Your DNA, including specific
genes

gene

(jeen):

The unit that passes heredity from parents to children. Genes are pieces of DNA and contain information for making a specific protein.

, contains instructions for making proteins that are essential for good health. Mutations, or changes in your DNA, can lead to proteins that do not work properly or that are missing altogether. These changes can cause genetic, or inherited, disorders such as cystic fibrosis, thalassemia, hemophilia, and sickle cell disease.

Genetic therapies are approaches that treat genetic disorders by providing new DNA to certain cells or correcting the DNA. Gene transfer approaches, also called gene addition, restore the missing function of a faulty or missing gene by adding a new gene to affected cells. The new gene may be a normal version of the faulty gene or a different gene that bypasses the problem and improves the way the cell works.

Genome editing is a newer approach that allows precise correction or other targeted changes to the DNA in cells to restore a cell’s function. Genome editing can:

• Remove a stretch of DNA that causes a disease
• Turn off a gene to prevent it from making a harmful protein
• Turn on a gene or instruct a cell to make more of a needed protein
• Correct a mutated gene

Gene transfer or genome editing treatments can directly modify the cells in your body, or your cells can be collected and treated outside of your body and then returned to you. For example, a doctor can remove immune system cells, cells that are part of your body’s natural defense system, or bone marrow cells from your body, modify their DNA, and then re-introduce them to your body.

The only genetic therapies that are currently approved by the U.S. Food and Drug Administration (FDA) are for a rare inherited eye condition, as well as certain types of cancer. Genetic therapies that are in development could treat or cure other inherited disorders; treat other cancers; or treat infections, including HIV.

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