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Novel Gene Editing Approach for Treating Cystic Fibrosis

A novel genetic-editing approach for the systemic treatment of cystic fibrosis (CF) is reported in Science Advances this week. While efforts to develop gene therapies for CF — a condition caused by mutations in the CF transmembrane conductance regulator (CFTR) gene — have been underway for years, they have all faced challenges delivering their therapeutic payloads to affected organs including the lung and gastrointestinal (GI) tract. Aiming to overcome this issue, Yale University scientists developed a non-nuclease-based approach to gene editing by using endogenous DNA repair stimulated by the binding of peptide nucleic acids (PNAs) to genomic DNA to create PNA/DNA/PNA triplexes that can initiate an endogenous DNA repair response. When PNAs are introduced with a single-stranded donor DNA containing the desired sequence modification, site-specific modification of the genome occurs. The researchers show that PNAs designed to correct a common CF-causing mutation can be delivered systemically using biocompatible polymeric nanoparticles into mouse models of CF, triggering a partial gain of CFTR function in both airway and GI tissues with no off-target effects. The work, the study's authors write, lays the foundation for systemic in vivo gene editing to correct CF.

The Scan

Team Tracks Down Potential Blood Plasma Markers Linked to Heart Failure in Atrial Fibrillation Patients

Researchers in BMC Genomics found 10 differentially expressed proteins or metabolites that marked atrial fibrillation with heart failure cases.

Study Points to Synonymous Mutation Effects on E. Coli Enzyme Activity

Researchers in Nature Chemistry saw signs of enzyme activity shifts in the presence of synonymous mutations in a multiscale modeling analysis of three Escherichia coli genes.

Team Outlines Paternal Sample-Free Single-Gene Approach for Non-Invasive Prenatal Screening

With data for nearly 9,200 pregnant individuals, researchers in Genetics in Medicine demonstrate the feasibility of their carrier screening and reflex single-gene non-invasive prenatal screening approach.

Germline-Targeting HIV Vaccine Shows Promise in Phase I Trial

A National Institutes of Health-led team reports in Science that a broadly neutralizing antibody HIV vaccine induced bnAb precursors in 97 percent of those given the vaccine.