Genetic medicine can leave people with rare mutations behind. But there’s new hope

Genetic medicine can leave people with rare mutations behind. But there’s new hope

26 Apr    AP, Finance News, PMN, PMN Business

Most of the 14 experimental gene therapies in the pipeline for the disease aim to help patients with any mutation, the Cystic Fibrosis Foundation says, delivering a new, correct version of the CFTR gene to cells. Getting correct copies of the CFTR gene would enable cells to make normal proteins no matter what mutation causes a patient to have no, or not enough, functional CFTR proteins.

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