Congenital, Hereditary, and Neonatal Diseases and Abnormalities: News and Research on Cystic Fibrosis

MeSH tree: C16.614.213

Cystic fibrosis: Causal treatment suitable from childhood

Cystic fibrosis remains an incurable genetic disorder that impairs lung function and significantly reduces life expectancy. A new combination drug therapy that addresses the disorder's underlying defects offers a promising ...

A new treatment approach for cystic fibrosis

Antisense oligonucleotides, or ASOs, are molecules that can be used to control protein levels in cells. Cold Spring Harbor Laboratory Professor Adrian Krainer leveraged ASO technology to develop the first FDA-approved treatment ...

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