Gene treatment is a medical technique that manipulates genes to treat and cure disease. It works by introducing genetic material into cells to compensate for abnormal genes or treat a genetic disorder. The introduced genetic material may provide a missing therapeutic gene or inhibit the activity of a mutated gene associated with disease.
Types of Gene Therapy
There are several types of gene treatment depending on the approach and goals. Some major types include:
Somatic Cell Gene treatment
This involves manipulating Gene Therapy in non-reproductive cells like liver, muscle and bone marrow cells to treat acquired diseases. Changes made through somatic cell gene treatment are not inherited by offspring. It is used majorly to treat cancers, monogenic disorders and acquired disorders.
Germline Gene treatment
Here, genes are manipulated in reproductive eggs, sperm or early embryo cells called germ cells. Any changes made will be passed onto offspring, making them permanent. It is mostly used experimentally and raises ethical concerns regarding unintended consequences.
Ex Vivo Gene treatment
In this method, genes are introduced into cells outside the body in a laboratory. Genetically modified cells are then transfused back into patients. It is commonly used to treat immunological, hematological and metabolic disorders.
In Vivo Gene treatment
With this technique, genetic material in the form of DNA or RNA is directly administered or delivered into a patient's cells and tissues through intravenous, intramuscular or other routes. It avoids cell removal and re-introduction steps of ex vivo therapy.
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