Satisfactory Essays

It was September fourteenth nineteen ninety in a hospital in Bethesda, Maryland. Doctor Culver was carrying a small plastic bag containing the first bag of genetically engineered cells intended to treat a human disease. SCID is a disease with a immune deficiency that can be controlled by gene therapy. This girl Cynthia was born with this disease SCID. She has been treated with gene therapy and so far it has worked. SCID is Severe Combined Immunodefficiency. It is a terrible disease that kids are born with and in order to get rid of it you need undergo gene therapy. It is a long process which is costly but well worth. The enzyme that these kids are not producing is ADA or adenosine deaminase. SCID is a birth defect where your parents have the recessive or broken down gene together. It costs a significant amount of money to get this treatment. It is about sixty thousand dollars for weekly injections of PEG-ADA. That is a drug that has the enzyme with a chemical sheath that allows it to get into the bloodstream. SCID is formerly known as the “bubble boy” disease. SCID is a defect in the white blood cells B- and T-lymphocytes that protect us from viruses and disease. A little more than half of the cases of SCID are inherited by the X chromosome from the mother. A mutation in JAK3 which is located on chromosome nineteen will make this defect happen (SCID). Also, X-linked SCID comes from a mutation in the interleukin 2-receptor gamma (IL2RG) gene which then causes SCID. These kids that are born with out a functional immune system can get pneumonia or

PG.2 chicken pox and can die before their first birthday. The first treatment ever done with this gene therapy worked and so did the second. Both of the kids Ashanti and Cynthia were not a hundred percent cured but enough for them to be able to live their lives regularly and be able to go places and play sports. They did have to get the weekly injections but it was a great success for the NIH people and all they had to do was get their child hood shots of the common disease and their injections so then they were fine. Yes it was a big hassle and very expensive but those kids were living regular lives for once in their life and they loved it and their parents were cherishing every moment of it.
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