What is Gene Therapy?
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Gene therapy and the techniques of genetic engineering in the treatment of a genetic disorder or chronic disease are still in experimental stages. There are two basic methods of Gene therapy- In vivo and Ex vivo Gene Therapy. While vivo method inserts genetically altered genes directly into the patient, the ex vivo method removes tissue from the patient, extracts the cells in question, and genetically alters them before returning them to the patient. So, one technique here involves removing cells from a patient, fortifying them with healthy copies of the defective gene, and reinjecting them into the patient and another involves inserting a gene into an inactivated or nonvirulent virus and using the virus’s infective capabilities to carry the desired gene into the patient’s cells.
The basic problem or challenge of gene therapy lies in development of a means to deliver the genetic material into the nuclei of the appropriate cells, so that it will be reproduced in the normal course of cell division and have a lasting effect. A liposome is also sometimes used to transport a gene into a body cell. A liposome is a tiny fat-encased pouch that can traverse cell membranes. Another approach employing liposomes, called chimeraplasty, involves the insertion of manufactured nucleic acid molecules (chimeraplasts) instead of entire genes to correct disease-causing gene mutations.
The drawback of this therapy is that the immune system may attack cells treated by gene therapy, that the viral vectors could mutate and become virulent, or that altered genes might be passed to succeeding generations.
Gene therapy was first used in humans in 1990 to treat a child with adenosine deaminase deficiency (ADA), a rare hereditary immune disorder (see immunity). In the United States, gene therapy techniques must be approved by the federal government.
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