HMB265H1 Lecture Notes - Lecture 22: Ex Vivo, Deoxyadenosine, Wild Type
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HMB265H1 Full Course Notes
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Corrects the diseased phenotype in affected somatic cells. A good target for gene therapy is a disease that only affects one gene: you only have to engineer one gene back in to recover the lost phenotype. Ex-vivo: remove affected cells > engineer > put in a gene that works > put cells back in. Only a portion of the cells need to be treated. Germ line therapy: genetically engineer germ line cells. Ethical issues are associated because the germ line can affect future progeny. Slide 4 - 7: first gene therapy trial in humans (1990-1992) Adenosine deaminase (ada) gene: unable to mount a normal response to infection. Deoxyadenosine > deoxyinosine -> > > uric acid (urine) The build-up of deoxyadenosine when ada enzyme isn"t working leads to a build up in deoxy atp > this destroys t cells and then they are unable to activate b cells, resulting in scid. One of the requirements of gene therapy:
