CF Therapy’s Effectiveness With Rare Mutations Can Be Determined in Lab

CF Therapy’s Effectiveness With Rare Mutations Can Be Determined in Lab

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CF Therapy’s Effectiveness With Rare Mutations Can Be Determined in Lab

An ex vivo technique using nasal epithelial cells from a young girl carrying a rare mutation profile for cystic fibrosis (CF) was successful at predicting the efficacy of an approved CF therapy, a case report shows. Researchers first tested the effectiveness of Orkambi (lumacaftor/ivacaftor) on the child’s own cells in the laboratory — a process referred to as ex vivo, meaning the cells were taken from a living organism and analyzed outside the body. As…

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