Gene Therapy
Gene therapy is a new and largely experimental branch of medicine that uses genetic material (DNA) to treat patients. Researchers hope one day to use this therapy to treat several different kinds of diseases. While rapid progress has been made in this field in recent years, very few patients have been successfully treated by gene therapy, and a great deal of additional research remains to be done to bring these techniques into common use.
Disease Targets
Humans possess two copies of most of their genes. In a recessive genetic disease, both copies of a given gene are defective. Many such illnesses are called loss-of-function genetic diseases, and they represent the most straightforward application of gene therapy: If a functional copy of the defective gene can be delivered to the correct tissue and if it makes ("expresses") its normal protein there, the patient could be cured. Other patients suffer from dominant genetic diseases. In this case, the patient has one defective copy and one normal copy of a given gene. Some of these disorders are called gain-of-function diseases because the defective gene actively disrupts the normal functioning of their cells and tissues (some recessive diseases are also gain-of-function diseases).
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