GENE THERAPY. What is gene therapy?. Gene therapy is the introduction of normal genes into cells that contain defective genes . Why gene therapy?. To supply the cells with the correct directions to make a missing protein product. How? 2 Delivery Systems. In vivo
Would this be considered in vivo or ex vivo?
Vectors are carriers. (ex. insects are considered vectors for pollen)
Vectors transport the DNA into the body or into the cells.
What wasthe vector in the transformation lab?
So, why not let the virus inject the good gene into a cell?
Imagine this lifecycle taking
place in the human body with
a good gene. Explain what
The Food and Drug Administration (FDA) has not yet approved any human gene therapy product for sale.
5) Target. One does not have control over where the gene will be inserted into the genome
4) Multigene disorders. Disorders such as heart disease, Alzheimer's disease, arthritis, and diabetes, are caused by the combined effects of many genes.General concerns
Major problems with gene therapy:
More than 5000 patients have been treated in last ~12 years worldwide
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