Gene therapy is a topic that has been researched more recently than most therapies for disorders. Genetic disorders like adenosine deaminase deficiency (ADA), or severe combined immunodeficiency (SCID), have been looked into for gene therapy, because of their single-gene mutation. These problems may be easily treated with gene therapy in the next few years of research. Children may be able to make it much farther than their original life expectancy, and may even eventually be able to live as a normal, healthy child.
Gene therapy is a technique that can treat and prevent diseases, specifically inherited diseases. It can be used to make a beneficial protein or bring in new material for mutated genes. There are certain techniques that are done …show more content…
William French Anderson at National Institute of Health started research on gene therapy in 1975, but eventually gave up for some time in the 80’s, because Anderson’s research did not lead anywhere, gene therapy was still a nonexistent method of the treatment. In 1984, Anderson decided to try again with gene therapy, with another specialist from NIH, Marshall Nirenberg. The specialists tried to focus on ADA, after figuring out that ADA only dealt with one gene mutation. Similar to other scientists’ methods for inserting new genes into the body, Anderson and Nirenberg used the idea of using a retrovirus to transfer new genes into a human. With all new ideas come new setbacks; for example, using a retrovirus can be very dangerous to the patient receiving treatment. One of the main concerns is safety, since retroviruses can cause cancer or AIDS; at the same time, retroviruses can only get to cells when they are dividing, so timing is of essence. Anderson and his colleague had many difficulties inserting these retroviruses into specimen, such as mice, monkeys, and human stem …show more content…
Jesse suffered from ornithine transcarbamylase deficiency (OTCD), which is where the ammonia that the liver does not break down, accumulates in the lungs. On September 13, 1999, Jesse was injected a virus that went to his liver. The virus started attacking his liver, and the ammonia levels started increasing. Each one of his organs starting failing, and Jesse eventually died on the 17th of September. The Food and Drug Administration decided to undergo an investigation, being that gene therapy is a relatively new technology.The FDA found that when Jesse Gelsinger came in, his initial ammonia levels were higher than normal, which should’ve made him ineligible for treatment. Prior to treatment, the consent form was not completed. Scientists did not mention that 2 patients before him that had liver damage suffered severe side effects while treated. After this incident, research on gene therapy slowed down, and not much was done for a
Gene therapy is a technique that can treat and prevent diseases, specifically inherited diseases. It can be used to make a beneficial protein or bring in new material for mutated genes. There are certain techniques that are done …show more content…
William French Anderson at National Institute of Health started research on gene therapy in 1975, but eventually gave up for some time in the 80’s, because Anderson’s research did not lead anywhere, gene therapy was still a nonexistent method of the treatment. In 1984, Anderson decided to try again with gene therapy, with another specialist from NIH, Marshall Nirenberg. The specialists tried to focus on ADA, after figuring out that ADA only dealt with one gene mutation. Similar to other scientists’ methods for inserting new genes into the body, Anderson and Nirenberg used the idea of using a retrovirus to transfer new genes into a human. With all new ideas come new setbacks; for example, using a retrovirus can be very dangerous to the patient receiving treatment. One of the main concerns is safety, since retroviruses can cause cancer or AIDS; at the same time, retroviruses can only get to cells when they are dividing, so timing is of essence. Anderson and his colleague had many difficulties inserting these retroviruses into specimen, such as mice, monkeys, and human stem …show more content…
Jesse suffered from ornithine transcarbamylase deficiency (OTCD), which is where the ammonia that the liver does not break down, accumulates in the lungs. On September 13, 1999, Jesse was injected a virus that went to his liver. The virus started attacking his liver, and the ammonia levels started increasing. Each one of his organs starting failing, and Jesse eventually died on the 17th of September. The Food and Drug Administration decided to undergo an investigation, being that gene therapy is a relatively new technology.The FDA found that when Jesse Gelsinger came in, his initial ammonia levels were higher than normal, which should’ve made him ineligible for treatment. Prior to treatment, the consent form was not completed. Scientists did not mention that 2 patients before him that had liver damage suffered severe side effects while treated. After this incident, research on gene therapy slowed down, and not much was done for a