Sentences with phrase «deliver normal genes»
The cystic fibrosis fix was elegantly simple: Deliver normal genes into the lung, and they would function in place of the faulty ones.
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The scientists are also experimenting with gene therapy, using a harmless virus to deliver a normal copy of the normal CIB2 gene to baby mice that have the mutated version.
The technique replaces the defective gene response for cystic fibrosis by using inhaled molecules of DNA to deliver a normal working copy of the gene to lung cells.
One approach is to deliver a working copy of the entire gene within a virus, but this method can disrupt normal genes.
Targeted gene therapies, however, had to wait for (1) the identification of the genes to target, (2) the cloning and / or sequencing of the relevant genes and in some cases, the specific disease - causing variant, (3) a full understanding of the normal gene function and regulation, and (4) the development of efficient ways to deliver genes to the relevant tissues at therapeutic levels.
A drug called Repoxygen delivers the EPO gene with some controls, so that when blood oxygen dips below normal, the body makes enough red blood cells to restore normal oxygen.
The modified virus then delivered the new genetic material inside the iPSCs, where the DNA region containing the broken gene was replaced with the sequence containing the normal gene.