The broad concept behind gene therapy is to replace defective
genes with working ones hijacking a virus as a delivery vehicle and
replacing its harmful genes with good ones. Need to engineer the
virus to be defective in some way so they can’t make more viruses/
modify it to carry “therapeutic genes”.
1) Look into the genome structure of a human retrovirus like
HIV and think of which genes you would like to mutate to make a
defective retrovirus suitable for gene therapy.
2) What kind of mutation would you engineer and where on the
selected genes?
3) Where would you insert the ‘therapeutic gene’? What other
regulatory sequences (if any) need to accompany this gene for
proper expression in the desired tissue?
Reading Frame 5′-LTR gag p24 p7 p prot 1000 2000 p51 RT 3000 tat nef l vif vou rev 3′ LTR p15 p31 int gp120 gp4 enV Vpr 4000 5000 6000 8000 7000 9000 9719 Base Pairs
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