Biology / Unit 7 Bio 1557 short lesson, about 20 seconds
Gene Therapy
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The idea
Gene therapy introduces a functional gene copy into patient cells, by a viral vector, to compensate for a disease-causing mutated gene.
In practice
A viral vector carrying a functional gene infects target cells and delivers that gene alongside the patient's own mutated copy.
Where people slip
Basic gene therapy adds a functional gene copy alongside the mutated one — it does not remove or repair the defect.
Your place on the trail
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