Topic Background

A little bit about Gene Therapy...

 The idea of gene therapy initially began around the 1960s to 1970s.  It was what scientists called "gene surgery", or using gene therapy to treat inherited diseases by changing defective genes.  This idea was considered for many diseases, one of the first being Lesch-Nyhan disease, a disorder caused by a deficiency of the enzyme hypoxanthine-guanine phosphoribosyltransferase (HGPRT) which leads to uric acid building up in all of one's body fluids.  The idea was born that one could inject an enzyme-producing gene for correcting this deficiency into a group of cells, and then these cells could be injected into people with Lesch-Nyhan disease.  It's almost in these simple principles that the mission of gene therapy lives.  There was only one problem though.  The promises for discovering new uses for gene therapy were choked by time taking ability to identify the genetic abnormalities that were the very cause of a certain disease.  On the other hand, a pick up in the interest of gene therapy lead to the growth of research.  For example, it was found that genetic defects in one or more genes occurred in successive generations of certain family members who suffered from a number of genetic disorders, and although these genetic defects might not be the cause of the actual disease, it could cause certain individuals to become more susceptible to developing the disease.  It could be triggered by environmental influences or things of that nature.

 In the year of 1990 a four-year old girl suffering from a rare genetic disorder underwent a process in which white blood cells were taken from her body, inserted with ADA producing genes, and then were transferred back into the body of the patient.  She had a weakened immune system and she was susceptible to severe infections, but the hope was that doing this process could help strengthen her immune system.  Although her production of ADA showed improvement, it was not a permanent fix and she had to keep having injections.

 Nevertheless, procedures like this jumpstarted a new era of gene therapy.  In this same year, patients suffering from melanoma were tested on with gene therapy.  The goal of this was to help patients produce antibodies to battle the caner, and experiments like these have begun the growing attempts of making gene therapy work.  Bringing all this history to the present, both DNA-based treatments and Cell-based treatments are being investigated.In 1991, the US government provided $58 million for gene therapy research.  Large companies are on the forefront and are hoping to make some breakthroughs in this new found research area.  Below is a video made by Cortical Studios that demonstrates how gene therapy could be used to deliver a specific healing gene to a liver cell.  This is just one of many examples of how gene therapy could be used to help heal the world.

 

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