Gene Therapy and Children
DNA controls most of the body’s basic functions needed to survive. But when there is an abnormality in a person’s DNA, genetic disorders, such as Down’s syndrome and color blindness, occur. Gene therapy is an experimental type of treatment meant to remedy genetic disorders.
According to the National Institute of Health, there are several approaches to gene therapy, including but not limited to:
- Replacing a mutated gene with a healthy gene
- Deactivating a mutated gene
- Inserting a healthy gene into the body
Gene therapy remains a valuable treatment option to those suffering from genetic disorders of any kind.
The first clinical trial for gene therapy was in 1990.
According to the Oak Ridge National Laboratory, not much progress has been made since then.
Several factors have kept experimentation with gene therapy from progressing:
- Healthy genes introduced / inserted into the body have to continue to work and remain stable for a long period of time. Many patients need gene therapy several times in order for the treatment to work effectively.
- The immune system attacks anything foreign introduced to the body. Additionally, if a patient needs gene therapy multiple times, the patient’s body develops a more efficient response to the foreign genes each time.
- Gene therapy usually uses viruses to carry genes into the body. Unfortunately with viruses, there is the risk that the virus will be able to cause diseases again once inside the body.
- Some genetic disorders affect not only one gene, but several. Diseases that involve several mutated genes are even more complicated to treat with gene therapy.
According to the Mayo Clinic and because gene therapy is still experimental, the only way to receive gene therapy is through a clinical trial currently. “The possibilities of gene therapy hold much promise. To date, however, that promise has not been realized,” said the Mayo Clinic staff in an online article.
According to the Mayo Clinic, gene therapy has had some success in treating immune deficiencies, hemophilia, hereditary blindness, and leukemia. In 2009, researchers in the UK were able to use gene therapy to destroy cancer cells in mice!
“We hope this therapy will be used to treat cancer patients in clinical trials in a couple of years,” said Dr. Andreas Schatzlein.
Although there is still much progress to be made in the realm of gene therapy, it remains a valuable treatment option to people who suffer from genetic disorders of any kind.