CMN Weekly (26 August 2022) - Your Weekly CRISPR Medicine News
Some of the best links we picked up around the internet
By: Gorm Palmgren - Aug. 26, 2022
Top picks
A paper in Molecular Cell describes the structure and engineering of the minimal type VI CRISPR-Cas13bt3 (also known as Cas13X.1). The structure revealed how Cas13bt3 recognises the guide RNA and its target RNA and provided insights into the nuclease's distinct activation mechanism. Furthermore, the authors rationally engineered enhanced Cas13bt3 variants and ultracompact RNA base editors.
A novel type V Cas12a endonuclease from Ruminococcus bromii (RbCas12a) can cleave target DNA templates efficiently, using the exceptionally high accessibility of PAM 5′-YYN and has a relatively wide temperature range from 20 °C to 42 °C. Furthermore, the Russian authors show that the human-optimised RbCas12a nuclease is also active in mammalian cells and can be applied for efficient deletion incorporation into the human genome.
Seelos Therapeutics has received an undisclosed amount in a grant from The Michael J. Fox Foundation to further its research in SLS-004 for treating Parkinson's disease. SLS-004 is a novel epigenome-editing approach to modulating the expression of the SNCA gene mediated by modification of DNA methylation using CRISPR-dCas9. In a rodent model, a single in vivo dose of SLS-004 has achieved a therapeutically desirable 27% reduction in SNCA mRNA and a 40% reduction in the expression of SNCA protein.
Verve Therapeutics will present preclinical data on its base editing therapeutic candidate VERVE-201 at the European Society of Cardiology 2022 Congress on Monday. VERVE-201 is designed to permanently turn off the ANGPTL3 gene in the liver, a key regulator of cholesterol and triglyceride metabolism, with a precise A-to-G base pair DNA change.
Point-of-care testing of Salmonella in foods is enabled with a new CRISPR Cas12a-based "sweet" biosensor coupled with a personal glucose meter readout. The indirect signal transformation from the original target gene invA to the final glucose signal was achieved through recombinase-aided amplification (RAA), CRISPR Cas12a reaction, enzymic reaction, and glucose signal reading. As a result, quantitative detection of Salmonella in spiked milk samples was achieved within the range from 1 to 1×103 CFU/reaction.