Orkambi® and amplifier co-therapy improves function from a rare <i>CFTR</i> mutation in gene-edited cells and patient tissue.

EMBO Mol Med · 2017 · PMC5582412 · PMID 28667089

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a positive trendno p-value reported

A similar treatment failed to increase CFTR channel activity in cultures derived from CF‐2 ( P = 0.06), although there was a positive trend.

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