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Gene Therapy

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

Retroviral Vector

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

Enhancement Gene Theory

you can use GT to make better people; make people more intelligent, etc.

Single-Gene Changes

mutations in one gene resulting in a change in its protein product or elimination of the protein altogether

Polygenic Disorder

mutations in more than one gene, sometimes environmental influences play in

Mitochondrial Changes

affecting many organ systems, these diseases are caused by mutations in mitochondria

Chromosomal Abnormalities

complete chromosomes or large regions of a chromosome are missing, duplicated, or modified in some way

Best GT Candidates

only a single gene causes the disorder, genes are identified and well-characterized

Ex Vivo

cells are removed from the body, gene is inserted, cells are returned to the body; no rejection because patient's own cells are used

In Vivo

gene is inserted directly into specific cells within the body using vectors such as viruses


can infect dividing and non-dividing cells; target respiratory tract; illegal


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.

$400 million

amount spent on research & development this year

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