pharmacologic

Genes are sections of DNA in your cells that are passed down (inherited) from your parents. You inherit two copies of each gene, one from each parent. Genes carry information that controls what you look like and how your body works.

There are about 20,000 genes that provide instructions for making proteins, which your body needs to work correctly.

Some genes don't make proteins themselves but instead help control how other genes work. They act like switches, turning other genes on or off. This means they help decide when, where, and how much of a protein is made in your body.

Changes in your genes are also called gene variants or mutations. These changes can be inherited, occur as you age, or result from environmental factors such as chemicals or radiation. Often, these changes have no effect, but sometimes, even a small change in the DNA can alter the instructions for making proteins. If genes don't make the right proteins, or don't make them correctly, this can cause a genetic disorder.

Gene therapy uses genes to treat or prevent disease by correcting genetic problems. It works by changing your genetic material, rather than relying on traditional treatments like medicine or surgery.

New genetic material can be delivered into cells in two main ways:

• ex-vivo treatment. Cells are removed from your body, modified by adding genetic material, and then placed back into your body.
• in-vivo treatment. Genetic material is delivered directly into your body, often through an injection.

A common form of gene therapy involves inserting a normal gene to replace an abnormal gene. Other approaches include:

• Repairing an abnormal gene
• Altering the degree to which a gene is turned on or off

Newer techniques offer different ways to correct genetic problems:

• Genome editing (also called gene editing). Instead of adding new genetic material into cells, this approach changes the DNA that's already in your cells. A well-known example is CRISPR-Cas9. This promising technique is still being studied and may soon be used to treat genetic disorders.
• Cell-based gene therapy combines gene therapy and cell therapy. An example of this would be CAR T cell therapy. Cells, which are often immune system cells, are genetically altered to help treat a disease and then introduced into the body.

Gene therapies are currently only approved to treat a small number of diseases, such as:

• Leber congenital amaurosis, an inherited eye disorder.
• Spinal muscular atrophy, a genetic muscle disorder.

While gene therapy holds great promise, it comes with risks and challenges. Studies are still ongoing to ensure these treatments are safe and effective. Some challenges include:

• Immune system reactions. Your body may see the new material as a threat and react to it.
• Unintended effects. Changes to DNA could affect other genes in harmful ways.
• High costs. Gene therapies are often expensive and are not widely accessible.

Medicines can treat diseases and improve your health. If you are like most people, you need to take medicine at some point in your life. You may need to take medicine every day, or you may only need to take medicine once in a while. Either way, you want to make sure that your medicines are safe, and that they will help you get better. In the United States, the Food and Drug Administration is in charge of ensuring that your prescription and over-the-counter medicines are safe and effective.

There are always risks to taking medicines. It is important to think about these risks before you take a medicine. Even safe medicines can cause unwanted side effects or interactions with food, alcohol, or other medicines you may be taking. Some medicines may not be safe during pregnancy. To reduce the risk of reactions and make sure that you get better, it is important for you to take your medicines correctly. You should also be careful when giving medicines to children, since they can be more vulnerable to the effects of medicines.

Over-the-counter (OTC) medicines are drugs you can buy without a prescription. Some OTC medicines relieve aches, pains, and itches. Some prevent or cure diseases, like tooth decay and athlete's foot. Others help manage recurring problems, like migraines and allergies.

In the United States, the Food and Drug Administration decides whether a medicine is safe and effective enough to sell over-the-counter. This allows you to take a more active role in your health care. But you also need to be careful to avoid mistakes. Make sure to follow the instructions on the drug label. If you don't understand the instructions, ask your pharmacist or health care provider.

Also keep in mind that that there are still risks to taking OTC medicines:

• The medicine you are taking could interact with other medicines, supplements, foods, or drinks
• Some medicines are not right for people with certain medical conditions. For example, people with high blood pressure should not take certain decongestants.
• Some people are allergic to certain medicines
• Many medicines are not safe during pregnancy. If you are pregnant, check with your health care provider before taking any medicine.
• Be careful when giving medicines to children. Make sure that you give your child the correct dose. If you are giving your child a liquid medicine, don't use a kitchen spoon. Instead use a measuring spoon or a dosing cup marked in teaspoons.

If you have been taking an OTC medicine but your symptoms don't go away, contact your health care provider. You should not take OTC medicines longer or in higher doses than the label recommends.

Food and Drug Administration