LMP299Y1 Lecture Notes - Viral Vector, Ex Vivo, Liposome

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Actual gene therapy use a gene as a therapeutic in genetic disease or acquired genetic disease. Correct the gene directing therapy at the root cause. Duration of effect for genetic disease, long-term effect. To achieve gene transfer generally, two roots one is the idea of ex vivo gene therapy; other is in vivo. In vivo directly insert the gene into the individual; hope that approach gets to the right kinds of cells. Ex vivo gene therapy good delivery vehicle ex. Turnover best target are stem cells, that can give rise to multiple other cells. Do not take tissue samples repeatedly from patient not desired. These issues apply to all types of gene therapy, all strategies. Most used method of delivery type of viral vector multiple kinds idea is that vector takes advantage of the very efficient gene transfer that tends to happen with viral infection .

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