In this study, we present a high-performance CRISPR-Cas ribonucleoprotein strategy to permanently correct the GPR143 mutation in these patient-derived iPSCs.
Our assembloid approach enables in a modular way to upgrade or exchange extraembryonic tissues to access more advanced stages of post-attachment development while complying with ethical policies.
Overall, these findings lend insights into molecular mechanisms underlying FHF regulation of INa,L in pathophysiology and outline potential therapeutic avenues.
Here, we detail recent advancements in CRISPR therapeutics to treat a wide range of autosomal dominant disorders and discuss how they are altering the landscape for future therapies.
A team of researchers from reports that they have essentially created a plug-and-play multi-organ chip, which is the size of a microscope slide, that can be customized to the patient.
This study demonstrates the remarkable robustness of the human replication timing program and reveals MCM10 as a novel candidate modulator of DNA replication timing.
