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the insertion of working copies of a gene into the cells of a person with a genetic disorder in an attempt to correct the disorder
Somatic Gene Therapy
uses patient's own cells to correct genetic disorders; pull cells, infect cells with gene, inject back into body
most commonly used in somatic gene therapy; viruses that only infect dividing human cells
Germline Gene therapy
permanent modifications of a gene in reproductive cells; can be passed to offspring
mutations in one gene resulting in a change in its protein product or elimination of the protein altogether
affecting many organ systems, these diseases are caused by mutations in mitochondria
complete chromosomes or large regions of a chromosome are missing, duplicated, or modified in some way
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only a single gene causes the disorder, genes are identified and well-characterized
cells are removed from the body, gene is inserted, cells are returned to the body; no rejection because patient's own cells are used
technique to introduce recombinant DNA into cells by applying a brief electrical pulse to a solution containing cells; the electricity creates temporary holes in the cells' plasma membranes, through which DNA can enter
cells bombarded w/ microscopic projectiles (made of inert substance like tungsten or gold) and coated w/ DNA, shot at high velocity from particle gun unto cells/tissues, ideal use in live organisms and in animal cells which lack cell walls-more easily transformed than plant/fungi cells. Viruses w/ affinity for certain cell types can be used as vectors if "loaded" with desired foreign DNA and allowed to infect target host cells.
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