BIMS 201 Lecture 12: adenosime deaminase deficiency
Document Summary
Individuals with ada deficiency have a form of s c id. S evere c ombined immune deficiency (s c id: not a singular disease, o ther forms of s c id can be inherited system, therapy. S econd therapy: g ene therapy where the good ada gene was put into the patient"s own white blood cells (ex vivo) and reintroduced into the patient. P e g -ada therapy was simultaneously performed: tagging protein with p e g helps to deliver it to the cells and protects against immune response to the cow ada protein, altering enough wbc was a problem. S urvivability of the wbc is a problem and the therapy had to be repeated. Longer lasting effects if used progenitor t cells! In bone marrow, accounts for 1 in 109 cells. S equences used to deliver the gene to the genome are often repetitive in the human genome. C ytokine genes will have certain amounts of homology.