Drug Development

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Western University
Anatomy and Cell Biology
Anatomy and Cell Biology 4429A
Paul Walton

If a protein is dominantlydefective it is hard to knock out the bad copy In RAS for example the mutant is always onputting a good copy of RAS into cells does not fix the presence of the bad over active copyMust be a drug that can be shipped around the world which will help everybody get itnot just a handful of people Viruses dont typically make enormous proteins so when trying to fix a gene that is large we will have problems A lot of retroviruses integrate wherever its convenient Integration is an enormously complex problem Some viruses only infect nondividing or dividing cells ex HIV is a retrovirus Retroviruses have single stranded RNA and have an enzyme called reverse transcriptase On the surface of the virus were envelope proteins that interacted with specific types of cells The envelope protein determined what type of cell the virus interacted with and infected The viral RNA integrates into the host cell DNA When the virus has integrated it uses host machinery to replicate Taken what we know about this and used it to therapeutic ends since around 1980Packaging cell is used for a retrovirus based vectornotice the simple retrovirus genome Want to isolate viral RNA and take away the viral genes and put in place my therapeutic genes The simple vector genome doesnt have a reverse transcriptase and targeting is a problem Because there is no viral gene they make viruses that dont have anything
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