Introduction of Gene therapy

Original Paragraph:

Gene therapy is likely to be most successful with diseases caused by single gene defects. The first successful gene therapy on humans was performed in 1990 by researchers at the National Institutes of Health. The therapy treated a four-year-old child for adenosine deaminase (ADA) deficiency, a rare genetic disease in which children are born with severe immunodeficiency and are prone to repeated serious infections.

Since 1990, gene therapy had been tested in human clinical trials for treating such diseases as severe combined immunodeficiency disease (SCID), cystic fibrosis, Canavan's disease, and Gaucher's disease. In 2003, more than 600 gene therapy clinical trials were under way in the United States but only a handful of these are in advanced stages. SCID, in which children lack natural defences against infection and can only survive in isolated environments, remains the only disease cured by gene therapy.

Key Ideas:

• Gene therapy is most successful with diseases caused by single gene defects.
• The first gene therapy on humans that was successful happened in 1990 in the National Institutes of Health
• The patient that received this treatment was a four-year-old child who had adenosine deaminase (ADA) deficiency, which is a genetic disease where children are born with critical immunodeficiency and are likely to have serious infections repeatedly.
• Gene therapy has been trying to treat diseases such as immunodeficiency disease (SCID), cystic fibrosis, Canavan's disease and Gaucher's disease since 1990.
• SCID is a disease where children lack natural defences against infection and can only live in lonely environments, which means that the only way it can be cured is by doing gene therapy.

Sources in MLA:

• Ama-assn.org, (2014). Gene Therapy. [online] Available at: http://www.ama-assn.org/ama/pub/physician-resources/medical-science/genetics-molecular-medicine/current-topics/gene-therapy.page [Accessed 7 Oct. 2014].

Paraphrasing:

  Gene therapy is most successful with diseases caused by single gene defects. The first gene therapy on humans that was successful happened in 1990 in the National Institutes of Health. The patient that received this treatment was a four-year-old child who had adenosine deaminase (ADA) deficiency, which is a genetic disease where children are born with critical immunodeficiency and are likely to have serous infections repeatedly. Gene therapy has been trying to treat diseases such as immunodeficiency disease (SCID), cystic fibrosis, Canavan's disease and Gaucher's disease since 1990. SCID is a disease where children lack natural defences against infections and can only live in lonely environments, which means that the only way it can be cured is by doing gene therapy.