Cystic Fibrosis Mechanism and Treatment | HHMI BioInteractive Video
2:31
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Cystic Fibrosis Mechanism and Treatment | HHMI BioInteractive Video
Mutations in the CFTR gene, which encodes a transmembrane ion channel, cause mucus buildup in the airways of patients with cystic fibrosis. This animation can be used to illustrate the connection between genotype and phenotype using a human disease example. It also demonstrates how knowledge about the genetic mutations responsible for a ...
303.9K viewsApr 19, 2018
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The journey to end cystic fibrosis isn’t a straight line. It’s an evolving map with many paths and unique challenges. That’s why we’re pursuing several different approaches to help achieve our goal of restoring CFTR protein and function in 100 percent of people living with cystic fibrosis. Dr. Bill Skach, EVP and Chief Scientific Officer at the Foundation, discusses how genetic-based therapies such as mRNA therapy, DNA therapy or gene therapy, and gene editing, could allow normal CFTR protein to
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