CONBOY LAB AND MURTHY LABResearchers aren’t just finding new applications for this precise and relatively easy-to-use gene-editing technique, they’re also tweaking it to give it new powers. Among this year’s developments:
RNA editing: A research team led by Feng Zhang of the Broad Institute fused an RNA-editing enzyme to an RNA-targeting Cas protein, enabling users to edit specific nucleotides within RNA molecules in human cells. The technique, called RNA Editing for Programmable A-to-I Replacement (REPAIR), is expected to help researchers investigate phenomena such as alternative splicing mechanisms and translation. The study’s authors suggest it could one day even be used therapeutically.
Base-editing human embryos: A team from Sun Yat-Sen University in China reported correcting, in living human embryos, the single-nucleotide mutation that leads to the blood disorder β thalassemia. The base-editing does not cut the DNA when it makes an edit, so it potentially has fewer harmful side effects than classic CRISPR-Cas editing would.
Nanoparticle delivery: ...