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.
Chinese researchers have developed a CRISPR-Cas12a-assisted assay for the ultrasensitive detection of pathogens. The method employs direct digital dual-crRNA (3D) and an adsorption-free self-priming digital chip. For example, the assay could directly detect Salmonella in milk without nucleic acid extraction.
Outlook on the Security and Potential Improvements of CRISPR–Cas9. This review focuses on the risks brought by gene editing therapy to the patient genome, which provides a broader perspective for exploring and improving the security of gene editing therapy from two aspects of delivery system and CRISPR editing tools.
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.