HMB265H1 Lecture Notes - Lecture 22: Cystic Fibrosis, Hematopoietic Stem Cell Transplantation, Ex Vivo

• School: UTSG
• Department: Human Biology
• Course: HMB265H1
• Professor: Maria Papaconstantinou

Document Summary

Introducing back normal genes can restore normal life for patients w/ genetic disorders. = therapy confined to somatic cells and not inherited. Gene therapy = delivery of therapeutic genes into body to correct disease conditions that arise from faulty genes. If loss of function, then only need to introduce normal gene back into cell. If gain of function allele, then need to inactivate bad gene/protein. If disease complex, might need more complicated approach. Maybe target genes that do cell proliferation. = what"s the nature of the disease, and then design gene therapy accordingly. Have to do w/o affecting normal gene function. Want stable expression of gene w/o interfering w/ normal gene function. Don"t want therapeutic gene to activate proto-oncogenes and turn them into oncogenes. [3] how can genes be directed to proper tissues/organs. Lungs filled with sticky mucus blocking airways = prone to infections. Loss of function of a gene = need to reintroduce back normal gene.