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L18 - *Gene Threapy-blood disease-planpotent stem...

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*Gene Threapy -blood disease -planpotent stem cells *Free DNA delivery disadvantages-Low efficiency and require culture, need cell that is actively dividing and need to select for them *Viral vector – advantage is high efficiency, disadvangatage need viral sequences (most commonly use retrovrisues like RNA viruses, RNA genome get copied into cDNA and get integrated), dividing or not, usually use diabeled viruses, these are viruses that lack the genes necessary to come back out and gets genes packaged. No coating protein. These viruses are replication incompetent means that you need something to replicate them. Helper virus allows for replication *Adenovirus- dsDNA- replicates in cells without integration, transient even with a competent virus. Gene therapy Examples -Attempt to cure patients -SCIDX-1 no immune system inactive gama cytokine gene, necessary for T-cell proliferation so patient does not have any immune system -2 infants (transformed bone marrow cells with an HIV based vector with the gama c cytokine gene in it.
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