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The treatment involved removing bone marrow from the infants, isolating the so-called stem cells, inserting normal genes to replace the defective ones, and then reinfusing the stem cells into the blood stream, where they were finally able to generate the cells for a functioning immune system.
This involves the insertion of a corrected gene to target cells to overexpress and replace the defective ones responsible for disease.
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They don't specify what exactly they'll replace the defective bits with, but I they'd be fools to replace them with similarly faulty parts.
Imagine buying two new tires for your car for $500, having one blow out, and the mechanic offering to replace the defective tire for $450.
Some are still holding out hope that they can eventually find a way to replace the defective dystrophin gene.
As of last week, 2,286 people had undergone a second operation to replace the defective hip sockets, Mr. Miller said.
All four carmakers have said that they will replace the defective parts for free.
Whereas drugs can only block or replace the defective proteins that cause genetic disease, gene therapy rewrites the DNA that contains the defective recipes.
If a hose is broken, pump cracked, etc., you will need to replace the defective item.
Gene therapy is focused on replacing the defective gene with a new one carried by different vectors; AAVs-mediated gene therapy for MDs is a feasible therapeutic approach that leads to the planning and implementation of phase I clinical trials.
Under the return policy, customers can send back the defective items after inspecting them and the seller purchases the defective ones at a lower price.
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