Italian researchers report the development of a base editing strategy to repair a mutation causing cystic fibrosis (CF). The 2789+5G>A CF transmembrane conductance regulator (CFTR) gene mutation causes an aberrant splicing and a non-functional CFTR protein. The method used a specific adenine base editor (NG-ABEmax) delivered as mRNA to patient-derived rectal organoids and bronchial epithelial cells. Base editing led to sufficient gene correction to recover the CFTR function while reducing bystander and off-target activities.
CRISPR-Cas-based methods for eliminating specific bacterial strains, such as antibiotic-resistant bacteria in the microbiome, are hampered by so-called escapers that mutate the plasmid-encoded Cas9 molecule and thus survive. However, Chinese researchers now show that the insertion of the transposable element IS5 is a common cause of Cas9 inactivation, and they have devised a strategy to overcome this problem. The two-fold solution is to eliminate the insertion process's genetics and increase the copy number of the Cas9 gene.
CRISPR - Genome editing in Medicine and Agriculture. This one-day conference on April 16 is held at the Weizmann Institute of Science, Israel will include 16-18 oral presentations of 15 minutes each covering advances in genome editing technologies as well as applications in medicine and agriculture.