RNAi, or RNA interference, prevents a defectivegene from making disease-causing proteins.
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TUSC2- defectivegene expression is detected in the majority of lung cancers and is associated with worse overall survival.
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It is caused by a defectivegene that allows secretions to block passageways in the lungs and pancreas.
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Methods: Genetic linkage analysis, exome sequencing, and functional genomics were carried out to identify and characterize the defectivegene.
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Many approaches based on gene therapy aim to repair the defectivegene or to insert genes with compensatory function.
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Mutation and linkage analyses have demonstrated the disease to be heterogeneous, with rBAT being the defectivegene in type I cystinuria.
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The therapy uses a virus to provide a normal copy of the SMN1 gene to babies born with a defectivegene.
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That is, unless you know that you carry a defectivegene that can produce a disease, and you choose to reproduce anyway.
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Vogelstein developed the method by fusing a specially developed strain of mouse cells with human cells that possibly contained a defectivegene.
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LRRK2 was previously identified as a defectivegene in Parkinson's disease, and it is also located in a risk region for Crohn's disease.
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In December, the field got its very first FDA approval with Luxturna, which corrects a defectivegene in a rare, inherited retinal disease.
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Through a simple blood test, medical science can now with absolute certainty tell any adult in this group whether they carry the defectivegene.
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The boys who received the treatment suffered from the early form of ALD, in which the defectivegene is found only on the X chromosome.
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At the outset, people with sickle cell trait were 57 percent more likely overall to have chronic kidney disease than people without a defectivegene.
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A defectivegene and its protein product cause the body to produce unusually thick, sticky mucus that clogs the lungs and leads to life-threatening infections.
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Scientists have used gene therapy to halt the progression of adrenoleukodystrophy, a fatal neurodegenerative disease caused by a single defectivegene, in two seven-year-old boys.