CF Foundation | RNA Therapy

Published on november 18, 2019 by

This video animation shows how ribonucleic acid (RNA) therapy might one day be used to treat cystic fibrosis. This therapeutic approach involves either repairing or replacing faulty messenger RNA that carries a copy of mutated genetic instructions to the protein-making machinery of the cell. These flawed instructions result in the production of a dysfunctional cystic fibrosis transmembrane conductance regulator (CFTR) protein, which causes cystic fibrosis. If the messenger RNA can be corrected or replaced, the correct version of the instructions would result in the production of a functional CFTR protein.


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