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.
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.
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.
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
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.
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.
Learn more about
therapy for Severe Combined Immune Deficiency syndrome (SCID) More
the United States, gene therapy techniques must be approved by the
federal government. More
of gene therapy. More
Somatic Cell Gene Therapy and Germ Line Gene Therapy
two "classes" of gene therapy More