PCL302H1 Lecture 8: Lecture 8 - Genes as Drugs I .pdf
28 Sep 2014
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What will we be talking about: treating disease by either replacing abnormal genes, or by modifying the expression of genes, approaches that generally involve the administration of dna, rna or related oligonucleotides, rather than classical" small-molecule drugs. Rnai therapy somatic gene replacement therapy growth factor therapy antisense oligonucleotide therapy suicide gene therapy. Modifications to sperm or egg cells that are passed to offspring as permanent alterations: no!! Eugenics practices aimed at improving" the genetic composition of human populations. This person suffering from hereditary defects costs the community 60,000. Categories of gene therapy: somatic gene replacement therapy. The introduction of whole genes into non- germ line cells to alter the function of those cells and thus the course of a medical condition. The boy in the bubble": adenosine deaminase (ada) deficiency, causes severe immune deficiency and repeated infections, first successful gene therapy: 1990 for ada.
