Dysfunction of the cystic fibrosis transmembrane conductance regulator (CFTR) — an anion channel found on epithelial cells of various tissues — causes cystic fibrosis and plays an important role in ...
Sionna Therapeutics, Inc. announced that preclinical data on its novel cystic fibrosis (CF) treatment candidates, SION-451 and SION-719, will be presented at the European Cystic Fibrosis Society’s ...
Artistic rendering of gene editing reagents — mRNA (red) and DNA (green and yellow) constructs — being packaged into a lipid nanoparticle (blue). UCLA researchers have developed a lipid ...
In a recent study published in the Nature Biomedical Engineering, a group of researchers optimized prime editing (PE) for the efficient correction of the cystic fibrosis (CF) transmembrane conductance ...
- ALYFTREK™ is approved for patients 6 years and older with at least one responsive mutation, including 31 additional mutations not responsive to other CFTR modulator therapies - - In head-to-head ...
Researchers have demonstrated that lipid nanoparticles can package and deliver an entire therapeutic gene along with gene-editing machinery - a combination of large, complex components that non-viral ...
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