
Gene Therapy Cures Immune System Defects
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Engineering, Biology, Health Sciences
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University
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Practice Problem
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Hard
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The video discusses Mustafa Hassan, a child born without a working immune system, who was successfully treated with gene therapy. This experimental technique repaired his inherited defect, allowing him to live a normal life. The process involves using a virus to deliver a healthy gene into bone marrow cells, which are then reintroduced into the patient. The therapy has been successful in 14 children, with only two failures, and has saved the NHS significant costs. Despite past concerns about cancer risks, improvements have made the therapy safer, and it is now considered part of mainstream medicine.
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OPEN ENDED QUESTION
3 mins • 1 pt
What were the previous concerns regarding gene therapy?
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3 mins • 1 pt
What is the current status of gene therapy in medicine according to the text?
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