Curing CF by gene therapy

Essay by sm_disney February 2005

download word file, 3 pages 4.7

Downloaded 34 times


Genetic engineering is used in the medical field to aid in research, produce drugs and medicines as well as a treatment of some diseases and disorders.

Cystic fibrosis (CF), a genetic disease, occurs when a defective gene produces thick, sticky mucus, which clogs the lungs, leading to lung infections. These also obstruct the pancreas, preventing digestive enzymes from reaching the intestines. Symptoms are salty-tasting skin, coughing, short breath, huge appetite yet little weight gain and bulky stools. Symptoms may vary due to the many mutations of the CF gene.

How CF gene therapy works?

One way to assess gene therapy in humans is using cold virus to put normal genes directly into damaged CF airway cells. Preferably, the healthy gene will be delivered through a vein.



· Gene therapy offers the best hope for a life-saving treatment by tackling the root cause of CF, rather than only treating the symptoms.

· It has the capability of producing a normal life for the individual that receives the therapy.

Gene therapy is a fast growing field of its own and more and more is learned about it every day, as technology is there to develop it.


· After the gene was in the patient, there is still the problem of getting it to go to the right spot on the chromosome. Most of the time, the healthy gene will insert itself randomly in the cell DNA. Depending on where the new gene inserts, since it is a random insertion, it can be harmful or helpful.

· When gene therapy can be actively used, it will be regulated to prevent unnecessary treatment that may be harmful to the patient.

· The time of effects of gene therapy was unpredictable.

· The technology being used now isn't...