Gene therapy has long promised to provide a solution for genetic blood disorders, but delivering on that promise has proven complex due to challenges including difficulties delivering the editing ...

You may have seen it in the news recently: a baby in Pennsylvania with a rare genetic disorder was healed with a personalized treatment that repaired his specific genetic mutation. The treatment was ...

The gene editing treatment targets the relatively common 1717-1G>A splicing mutation, which does not respond to many current ...

ABE 8 variants show a higher editing efficiency, but a larger editing window. ABE7.10 shows the opposite: a smaller window, but lower efficiency. The trajectory of base editing has been remarkable, ...

In a global first, scientists have used advanced base editing to repair a deadly gene mutation in a newborn, marking a major leap forward in the treatment of rare ...

CyDENT represents a revolutionary advancement in genetic modification, facilitating CRISPR-free base editing within mitochondria and chloroplasts. This breakthrough technology heralds a new era in ...

Forbes contributors publish independent expert analyses and insights. Juergen Eckhardt leads Bayer’s impact investment unit, Leaps by Bayer. Dr. David Liu, pictured with former lab members Holly Rees ...

Scientists at St. Jude Children’s Research Hospital report that they have developed an unbiased, sensitive, and resource-efficient method to identify small, off-target sites that pose a safety risk ...

A schematic showing the cytosine base editing intermediate and the various outcomes that can occur when the UNG protein is active. You may have seen it in the news recently: a baby in Pennsylvania ...

Point mutations in the lamin A (LMNA) gene cause several human diseases, ranging from congenital muscular dystrophy to premature aging, collectively known as laminopathies, which affect skeletal ...