gene therapy

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Last updated 3:50 PM on 3/29/26
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5 Terms

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gene therapy

addition of a healthy gene to replace a faulty gene to treat a disorder

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types of gene therapy

somatic cell gene therapy- replaces faulty alleles with healthy alleles in body/somatic cells

germ line cell gene therapy- inserting healthy into early embryos or gametes

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differences between somatic cell gene therapy and germ line cell gene therapy

[using s and g btw]

-S inserts genes into body cells

-G inserts genes into embryo

-S only some cells get functional gene [targets specific cells]

-G all cells get functional gene

-S is short-term/ needs repeating

-G is long-term/ does not need repeating

-S cannot be passed to offspring

-G can be passed to offspring

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how is gene inserted into cell during gene therapy?

using vectors e.g. viruses, plasmids, or liposomes

→transfers healthy gene into cell

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PROS and CONS of gene therapy

add g and s

PROS

-extends life span

-enhances quality of life

-long-term cure [g]

-offspring will inherit healthy gene so won’t inherit disease [g]

CONS

-require multiple treatments as cure is temporary [s]

-virus vector may cause disease

-only helps the treated individual, not offspring→ offspring could still inherit disease [s]

-alleles could be inserted in incorrect place and could lead to mutation or cancer

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