HMB265H1 Chapter Notes - Chapter 22: Gene Therapy, Cystic Fibrosis, Retrovirus

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

Document Summary

Hmb265 lecture 22 readings - human gene therapy. Change genomes of germline cells = create stable lines of experimental organisms. Changing human gene where changed gene transmitted to next generation = ethical problems. = focus on changing genomes of somatic cells in patients. Gene therapy: introduce therapeutic gene into patient"s somatic cells. But need diff genes for diff diseases. Molecular nature of disease = what type of gene used for therapy. Disease caused by loss of function in gene. Therapeutic gene = wt copy of gene. Therapeutic gene inactivates disease gene or protein product. Target gene/protein from biochemical pathway that leads to cell proliferation. First need to choose what therapeutic dna to use. Then need to deliver therapeutic dna into somatic cells. Ex: in congenital blindness need to to introduce dna into retinal cells. Ex: in cf patients, need to introduce dna into lung cells. Can access tissue easily, but cells can"t be removed from patient"s body.