Skip to main content
Premium Trial:

Request an Annual Quote

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

Positive Framing of Genetic Studies Can Spark Mistrust Among Underrepresented Groups

Researchers in Human Genetics and Genomics Advances report that how researchers describe genomic studies may alienate potential participants.

Small Study of Gene Editing to Treat Sickle Cell Disease

In a Novartis-sponsored study in the New England Journal of Medicine, researchers found that a CRISPR-Cas9-based treatment targeting promoters of genes encoding fetal hemoglobin could reduce disease symptoms.

Gut Microbiome Changes Appear in Infants Before They Develop Eczema, Study Finds

Researchers report in mSystems that infants experienced an enrichment in Clostridium sensu stricto 1 and Finegoldia and a depletion of Bacteroides before developing eczema.

Acute Myeloid Leukemia Treatment Specificity Enhanced With Stem Cell Editing

A study in Nature suggests epitope editing in donor stem cells prior to bone marrow transplants can stave off toxicity when targeting acute myeloid leukemia with immunotherapy.