Using CRISPR and other tools, scientists are modifying DNA methylation, histone marks, and other modifiers of gene expression to understand how they affect health and disease.
Doctors in Oregon delivered the gene editing machinery behind the retina in hopes of treating an inherited form of blindness, according to the companies that developed the therapy.
Tips on how to surmount the challenges of working with CRISPR to manipulate genes in human stems cells to study their function in specific diseases or to correct genetic defects in patient cells.