Human Gene Therapy

Human gene therapy

Gene therapy is a technique used in attempts to cure or prevent genetic diseases at the molecular level (basically at the source) by correcting what is wrong with defective genes, a good version of the defective gene is introduced into the existing cells. This technique is still considered experimental, only being done through clinical trials. This idea was first suggested during the 1950’s. The basic idea was “if the basses can be arranged incorrectly then why can’t they be rearranged in the correct way?”
A genetic disorder is a disease that is caused by a mutation (change in DNA sequence that makes up a gene) in a person’s DNA. Inheriting a faulty gene can cause susceptibility to cancer and can cause many different conditions. A mutated gene creates a defective protein, which results in a disease (Defective gene▬►defective protein▬►disorder). Since gene therapy targets the faulty gene it treats the actual disease instead of treating the symptoms like most current medicine and treatments. Examples of genetic disorders are phenylketonuria, Huntington’s disease, sickle-cell anemia, cystic fibrosis and polycystic kidney disease. Gene therapy can also treat acquired diseases like some cancer, and Parkinson ’s disease. In 1990 adenosine deaminase deficiency (the lack of an important enzyme in the immune system) was the first genetic disorder to go through gene therapy trials. It was chosen because a simple gene causes it. The promising results gave gene therapy a good and hopeful start.
Gene therapy is a relatively simple process. First, the mutated gene at fault for the cause of the condition being treated must be identified. Second, the site of the unhealthy cells in the human has to be found. Then, a health...

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http://articles.latimes.com/2012/sep/13/health/la-he-future-of-gene-therapy-20120913