Genes are the basic units of heredity. Human genes contain information that tells the body how to work. They are responsible for cellular structure and function. When a gene is defective, it prevents the cell from carrying out its normal functions.

What is human gene therapy?

Human gene therapy involves putting genetic material into the body to correct faulty or missing genes. Gene therapy is used to treat or cure disease and health abnormalities.

What conditions can be treated using human gene therapy?

Gene therapy could potentially be used to treat cancer, cystic fibrosis, heart disease, hemophilia, wounds, and AIDS.

What agency regulates human gene therapy products?

The Food and Drug Administration (FDA) regulates drugs, medical devices, and biological products, assuring that they are safe and effective before they can be used. The FDA's Center for Biologics Evaluation and Research (CBER) regulates human gene therapies. Gene therapy research is experimental at this stage, and no gene therapy product has been approved for sale. The FDA oversees research in the area of gene therapy. The FDA temporarily halted gene therapy trials being conducted on children who had "bubble baby syndrome" as a result of severely compromised immune systems. Two children involved in the testing had developed a leukemia-like condition.

What are the recent developments in human gene therapy research?

In 2002, scientists successfully treated sickle cell anemia in mice. In 2003, scientists were able to transfer genes into the brain. This accomplishment could lead to treatment for Parkinson's disease. Researchers have also developed a therapy that might treat Huntington's disease.

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