Technology: Currently, there are only two approved therapies for Idiopathic Pulmonary Fibrosis (IPF), pirfenidone and nintedanib, which are minimally effective at slowing the disease. These treatments are not effective at all in halting or reversing the progression of IPF. Therefore, there remains a.....
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Genetic silencing avoids off-target effects of small molecule inhibitorsGene-specific silencing opens opportunity to discover disease mechanism
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Organism-wide frataxin knockdown (up to 90%)Inducible: able to control the onset and progression of the diseaseReversible: able to reverse the acceleration of disease progressionExhibit various symptoms parallel to FRDA patients
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