MICR3002 Lecture Notes - Lecture 1: Severe Combined Immunodeficiency, Phagocytosis, Transfection
Document Summary
Technique for introducing the genetic material of a gene in a patient in which the original gene is dysfunctional because of a mutation or deletion: correcting the gene. Strategies: ex vivo, cells removed from body, transgene delivered, cells cultured, cells returned to the body. In vivo: transgene delivered directly to the host. Vectors are used to insert the gene into a patient: most common vectors are viruses, account for ~67% of all gene therapy trials) Why viruses: viruses have evolved to infect cells with great specificity, can deliver dna/rna into the host genome with high efficiency (due to high replication) Remove viral sequences responsible for pathogenicity (and replication for retroviruses) Separate viral sequences required for replication and protection. Flank transgene by essential cis-acting sequences and packaging signals. Provide viral proteins required for packaging (and replication) in packaging cell line: some vectors retains replicative genes. Manipulation of virus is specific to the individual virus.