Improving the Nation’s Nutrition and Health

Curing Genetic Diseases Prior to Birth

Issue (Who cares and why?)
     You have just received the results of your prenatal screening and discovered that your unborn child has a life-threatening disease that will require lifelong therapy, but will not result in a cure, since the disease will cause a great deal of damage to the child during development, prior to birth. Your doctor gives a single injection of a genetically engineered virus containing a normal copy of the DNA that your child is lacking, and your child is born normally and lives a full healthy life, requiring no further treatments. This miracle treatment is known as fetal gene therapy, and a team at the University of Nevada, Reno has spent the last 12 years delineating the conditions that will one day make this dream a reality.

What has been done?
     The UNR researcher’s original goal was to determine whether it was possible to perform gene therapy in the fetus, early enough in development to permit delivery of the necessary genetic material prior to the onset of the irreparable tissue damage that takes place in many genetic diseases. A method was developed in which a single injection of a defective virus that had been engineered to carry a piece of “marker DNA” in place of its own viral DNA was given to young sheep fetuses, and researchers found that this approach resulted in lifelong presence of cells containing the marker DNA within the sheep. Even more exciting was the finding that this DNA was being expressed and made into protein that, in the case of a patient with a genetic disease, could result in correction of the disorder. This protein was expressed in numerous organs of the sheep, suggesting that this simple approach could one day be used to treat genetic diseases that affect essentially any tissue in the body.

Impact
      Once optimized and tested in a model system that accurately mimics a specific human disease, this type of therapy is one of the only treatments that could promise to offer a precise and specific means of permanently curing a wide range of genetic diseases before birth, thereby allowing the birth of a healthy infant who would require no further treatments.

Contact
Christopher D. Porada
Animal Biotechnology/ MS202
University of Nevada, Reno
Reno, NV 89557-0104
cporada@cabnr.unr.edu