PHYS30005 Lecture Notes - Lecture 22: Exon, Reading Frame, Myotonic Dystrophy

• School: University of Melbourne
• Department: Physiology
• Course: PHYS30005
• Professor: Charles Sevigny

Document Summary

Lecture 22 new therapies to improve muscle. Gene therapy = delivery of dna/rna to correct or compensate. Correction restore expression of a gene that is missing or reduced and/or reduce expression of a gene that is excessive or inappropriate. A delivery tool deliver a genetic element to cells or deliver a genetic element into a human. Bottom-up approach - sta(cid:396)t (cid:862)si(cid:373)ple/safe(cid:863), e(cid:374)ha(cid:374)(cid:272)e effi(cid:272)ie(cid:374)(cid:272)(cid:455: naked dna/rna fragment or plasmic, chemical modifications, targeting ligands/conjugates, lipid encapsulation. Top-down approach sta(cid:396)t (cid:862)effe(cid:272)ti(cid:448)e(cid:863), add safet(cid:455) a(cid:374)d (cid:272)apa(cid:271)ilit(cid:455: virus-based vehicle (evolved for delivery, remove viral genes/pathologic properties, modify/combine virus attributes. A viral vector has the key viral genes removed and replaced with good genes. Different viruses make different viral vectors for different delivery platforms. The two main viral vector platforms used are lenti- and adeno-associated viral vectors. Lentiviral vectors for gene delivery to muscle: deliver to muscle progenitor cells (satellite cells) or to muscle and then to stem cells, used for blood based diseases.