Before we get into what Gene Therapy is, we need to understand what genes really are! Click HERE to learn more about genes!

Scroll down below to read on ahead about the History of Gene Therapy!

History of Gene Therapy:

In the early 1970’s scientists began to propose an idea for treating inherited diseases that are caused by abnormal genes which was called “gene surgery”. The purpose of proposing this idea was to be able to take out the disease-causing gene, and then surgically implant a certain gene that functions properly. However, due to the lack of biological knowledge, scientists needed much expertise to perform this type of surgery on the human body. Around 1983, scientists from Baylor College of Medicine in Houston, Texas,proposed that gene therapy can be helpful for treating the Lesch-Nyhan disease. Experiments were made by injecting an enzyme-producing gene into a group of cells for replication, so that it may correct the disease. With the experiments, scientists then speculated that by injecting cells into people who carry the disease, it can correct the genetic defect that caused the disease to begin with. Throughout the 1980’s, as the science of genetics progressed, medical scientists believed that gene therapy could be a promising method for treating genetic based diseases.Due to the fact that scientists were able to identify specific genetic defects that cause inherited diseases, the growth and research of gene therapy escalated.

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Click on the picture to view it on a larger version from the original source!

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Before and after pictures of Ashanti Desilva. On the left, Ashanti is with Dr.W.French Anderson. On the right, this is Ashanti after the procedure. She is all grown up now.

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 On September 14,1990, the first person to undergo the process of gene therapy was a four- year old girl, Ashanti DeSilva, who was suffering from a genetic disorder that prevented her body from producing the enzyme: adenosine deaminase (ADA).This caused her body to have a weak immune system, making her extremely vulnerable to both minor and dangerous infections. Dr. W. French Anderson of the National Heart, Lung and Blood Institute, and Dr. Michael Blaese and Dr. Kenneth Kulver, both from the National Cancer Institute, performed gene therapy on Ashanti at the National Institutes of Health’s Clinical Center in Bethesda, Maryland. They took white bloods cells (which are necessary for the immune system to function properly) from the girl, and inserted ADA producing genes into the white blood cells, which they then transfused the cells back into Ashanti. As a result, Ashanti’s cells were able to produce the enzyme ADA.

 

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