What Is Somatic Gene Therapy?
Somatic gene therapy is a type of gene therapy that introduces a functioning gene into cells of an affected person that are not produced by sperm or eggs. This type of gene therapy can cure genetic diseases without the risks associated with germ line gene therapy.
Therapeutic genes can be introduced into cells through viruses, bacteria or plasmids (molecules containing DNA). These insertions, commonly referred to as “transgenes,” enable the production of protein or enzyme that has beneficial effects on the body.
It is essential to remember that somatic cell gene therapy differs from conventional gene therapy in that the genes introduced are only active within the cells they are injected into, and cannot prevent mutations from being passed on to future generations.
In the United States, FDA requires sponsors of somatic gene therapy trials to obtain informed consent from patients before enrolling them in the study. This is done to guarantee patients are informed about all potential risks, benefits and side effects associated with treatment.
Somatic gene therapies are typically delivered via viral vectors containing the therapeutic gene or product; however, nonviral vectors can also be utilized.
Experimental forms of somatic gene therapy are being explored for treating genetic diseases, such as sickle cell anemia. In one experiment, researchers used a gene responsible for the disorder to create a protein that could restore normal blood cell production.
Other approaches to somatic gene therapy are being researched, such as the delivery of proteins through muscle cells. In this technique, a recombinant protein that is secretory and without detectable toxicity is introduced into skeletal muscle tissue through a signal sequence linking it to the endoplasmic reticulum.
Proteins can be delivered in the form of a liposome or other carrier molecules. Encapsulating materials like beads or fibers may also be utilized to facilitate their delivery and biological activity.
Somatic gene therapy is a novel and promising treatment for many diseases that had previously been untreatable. Its potential has been recognized by both industry and government agencies alike, leading to significant investment in research and development efforts.
Somatic gene therapy is a technique designed to deliver therapeutic genes into specific cells within an individual’s body, such as those producing blood or proteins that affect immunity. The purpose is not just to mask a genetic disease but rather eliminate it completely.
Somatic gene therapy can be conducted in either a laboratory or clinical setting, depending on the patient’s specific condition and desired outcome. In the U.S., this technique has become increasingly popular and is overseen by the Food and Drug Administration’s staff who monitor both safety research and clinical trial outcomes.