Gene Therapy




 Medicine- Gene Therapy

Some of the most promising and powerful applications of genetic engineering are in the field of medicine. Researchers are using it to diagnose and predict disease, and to develop therapies and drugs to treat devastating diseases like cancer, Alzheimer's, diabetes, and cystic fibrosis. Explore more about one of the common uses of genetic engineering in medicine; gene therapy.

Gene therapy can theoretically be used to treat, cure or even prevent disease. Gene therapy is still very experimental and controversial, with some stunning success stories and some devastating failures attributed to its use.

Gene therapy can be used to fix defective genes or to replace missing genes. Many diseases are the result of just one gene malfunctioning; sickle cell anemia, cystic fibrosis, SCID, are all caused by one defective gene. To correct the problem, gene therapy is used to deliver genes that function correctly.

Other diseases are the result of a missing gene; juvenile Paget's disease - an extremely rare bone metabolism disorder - is one example. In those cases, gene therapy can be used to deliver genes to replace the missing one.

Just like a mailtruck delivers mail, or the UPS truck delivers packages, there has to be a way to deliver the good genes to the right "address." There are a number of ways to deliver the genes used in gene therapy. Sometimes the genes are packaged in a virus, they can be attached to a protein, or they can be encapsulated (like a pill). These different delivery methods aren't always effective or reliable. Delivery problems are one of the main reasons gene therapy is still so experimental.

The Future
Current uses of gene therapy focus on treating or curing existing conditions. In the future, the focus could shift to prevention. As more of the human genome is understood, medicine will know more about which genes contribute to or cause disease. With that knowledge in hand, gene therapy could be used to head off problems before they occur.

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Learn more about gene therapy.

Explore More: Genetic Engineering
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Gene Therapy
Gene therapy for Severe Combined Immune Deficiency syndrome (SCID) More

Who Oversees the Research?
In the United States, gene therapy techniques must be approved by the federal government. More

Delivery Disruptions
Obstacles of gene therapy.

Somatic Cell Gene Therapy and Germ Line Gene Therapy

The two "classes" of gene therapy More