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Discovered that genes can be transfered from one organism to another.
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Viruses can now be used to tranfer genes
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Gene therapy was first attempted on a human
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The laboratories in the National Heart, Lung, and Blood Institute and the National Cancer institute worked together to prove that cells with ADA defiency can be corrected in tissue culture. A retrovirus was used to carry the correct human ADA gene to the cells.
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Drs. W. French Anderson and MIchael Blease and their team study the safety and effiency of correct genes that were transfered into bone marrow cells into animals. Although the process wasn't harmful, the number of cells that receieved the correct gene was too small to be useful.
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The researchers decided to use white blood cells instead of bone marrow cells. This switch increased the number of correct genes taken up by the cells in the animal experiements. The success of these experiments led the researchers to start to look to due testing on people.
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Steven Rosenberg worked with a team to grow tumor infiltrating lymphocytes (TIL cells) from people with the deadly cancer malignant melanoma and then they engineered a virus to put DNA marker into those cells. These "marked TIL cells" helped researchers which TIL cells work best for cancer treatment and that the engineered virus can be used safely in humans.
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Drs. Anderson, Blease, and Kenneth Culver used a virus to deliver the correct ADA gene to a four-year old girl and a nine-year old girl with ADA Deficiency. Each girl was given repeated treatments over a period of two years.
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Gene therapy researchers were granted $58M for their research by the United States government.
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Researchers use gene therapy to treat newborn babies with ADA deficiency becasue their normal ADA genes were delivered to immature blood cells isolated from the babies' umbilical cord.
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An 18 year-old boy named Jesse Gelsinger dies becasue the therapy did not succeed in curing is liver disease.
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London Moorfields Eye Hospital has continued successful trials in curing blindness and failing eyesight.