Why are diseases of the blood simpler targets for treatment by ge...

Question

Why are diseases of the blood simpler targets for treatment by gene therapy than are many other genetic diseases?

Accepted Answer

Welcome back, everyone. Here's our next question. It says gene therapy is a medical technique that involves introducing genetic material into a patient's cells to treat or prevent disease. It has the potential to treat a variety of genetic disorders, cancers and blood diseases. Which of the following statements regarding gene therapy is correct. Choice. A gene therapy involves repairing the defective gene in the patient's cells. Choice B, it involves replacing the defective gene with a functional copy. Choice C and involves modifying the patient's immune cells to attack the defective gene or Choice D. It involves removing the patients affected cells and replacing them with healthy cells. When we talk about gene therapy, uh we're generally talking about Choice B replacing the defective gene with a functional copy. When we look at our other answer, choices. Uh Choice A which has repair, it involves repairing the defective gene. Um We just don't have the technology to do that very effectively yet. So that's not used. Instead, the whole gene is replaced. Looking at choice C involves modifying the patient's immune cells to affect the defective gene. Uh This is not commonly used. It could be done, but it's not the commonly used approach. Instead, it's a more direct replacement of the defective gene. And then finally, so that's why choice C isn't correct. And then finally, with choice D involves removing the patient's affected cells and replacing them with healthy cells. Um this is just too difficult um to target specific cell types. So that's why that's not our correct answer here. So we can rule out choice D. And again, our answer here as to the true statement about gene therapy, choice B gene therapy involves replacing the defective gene with a functional copy. See you in the next video.

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Why are diseases of the blood simpler targets for treatment by gene therapy than are many other genetic diseases?