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1) Indicate which of the following diseases could be corrected by directly infecting the affected cell type with a transgene as opposed to grafting:
a) an individual that lacks expression of an enzyme due to a recessive mutation within the gene.
b) an individual with degenerating dopaminergic neurons.
c) an individual with atherosclerosis.
2) Indicate what viral vector or transfection method can be used for gene transfer into the following primary tissues:
a) Fibroblasts
b) Neurons
c) Hepatocytes
d) Lymphocytes
3) Why is the Lac Z gene encoding b-galactosidase used in gene transfer studies?
4) List 3 ways to limit transgene expression to a specific cell type in gene therapy.
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