HMB265H1 Lecture 20: Gene Therapy (Lecture 22)
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HMB265H1 Full Course Notes
Verified Note
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Document Summary
Gene therapy is potentially one of the most promising treatments lately emerging. Somatic gene therapy: non-heritable engineer somatic cells to correct mutations that have happened within those cells in ex. First gene therapy trial in humans (1990 to 1992: severe combined immunodeficiency disease (scid, caused by mutation in adenosine deaminase (ada) gene. This gene is involved in the development and maintenance of the normal immune system. Effect of a defect in the ada gene. Destroys cells that are critical for the immune system: ada helps the metabolic pathway continue it converts deoxyadenosine into deoxyinosine which continues down the pathway to become urine. In absence of the ada gene the. Deoxy atp: this deoxy atp builds up and destroys t-cells; and b-cells are inactive. The protocol involved 2 girls age 4 & 9 years old: before, during, and after trial treated with peg-ada, the girls had some t cells so they didn"t have the severe form of the disease.
