PHRM3041 Lecture Notes - Lecture 2: Immunogenicity, Retrovirus, Oncogene
Document Summary
An experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient"s cells instead of using drugs or surgery. Approaches to gene therapy: replacing a mutated gene that causes disease with a healthy copy of the gene. Inactivating, or knocking out a mutated gene that is functioning improperly. Introducing a new gene into the body to help fight a disease. Mutations in cystic fibrosis transmembrane conductance regulator (cftr) gene. Chloride channel in apical membrane of exocrine epithelial cells. Respiratory, gi, reproductive tracts obstruction by thick viscous secretions. Current approaches of treatment: antibiotics, anti-inflammatory, mucolytics, nebulized hypertonic saline. Could create a therapy to correct the channel defect. Genetic material has to be delivered across the membrane and to the nucleus. We need to have some efficient means of delivering the genes and regulating their expression in our host system (human or cell system)
