Human Gene Therapy Essay

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Human Gene Therapy

Image what it would be like if doctors could cure Huntington's disease, muscular dystrophy, or even hemophilia. Could this be possible? With gene therapy this all could be reality in the near future. Gene therapy is a potential approach to the treatment of genetic disorders in humans. This is a technique where the absent or faulty gene is replaced by a working gene, so the body can make the correct enzyme or protein and consequently eliminate the root cause of the disease (BIO, 1990).

There are two types of gene therapy treatment: Somatic cell gene therapy and germline therapy. Somatic cell gene therapy involves obtaining blood cells from a person with a genetic disease and then introducing a normal gene into the
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And also how do you make the delivery specific and safe? The first step in gene therapy is an accurate diagnosis of the genetic defect. This is done by using a DNA probe. The DNA probe is specific to a complementary piece of DNA. This technique using DNA probes is more specific then other conventional methods of diagnosing genetic defects in humans (BIO, 1990). After the correct diagnosis is made, healthy DNA can be inserted into a virus, which has had infectious genes stripped out of it. The virus is then mixed with cells taken out of a patient and then injected back into that patient. Viruses are used because they are like genes, but wrapped in a special protein coat. On the surface of this protein coat are specialized proteins that bind to the surface of cells. Once these viruses are in the body they lock in place on specific cells. The cells then suck the viruses in or the viruses force their way into the cells (PBS).

A retrovirus is a type of virus that is used in gene therapy. This is a virus that inserts its genetic code directly into the chromosomes of the host cell (PBS). Other viruses are used for different kinds of genetic problems. One of these is the adenovirus which is used for cystic fibrosis patients (Coutts, 1998). To use a virus in gene therapy, the patient's immune system has to be weakened so it will not fight off the virus. If weakening the patient's immune system is not
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