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

X-linked chronic granulomatous disease (X-CGD) is a rare genetic disorder characterized by high susceptibility to infections that results from genetic mutations in the cytochrome b-245 beta chain ...

In 2025, baby KJ Muldoon became the first person to receive a personalized gene editing treatment, which likely saved his ...

The promise of genome editing to help understand human diseases and create new therapies is vast, but technological limitations have limited advancement of the field. While existing editing ...

Beam Therapeutics has presented the first clinical data on its base editing technology. All four sickle cell disease patients in the efficacy cohort had fetal hemoglobin levels above Beam’s target—and ...

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 ...

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 ...

Beam Therapeutics’ experimental gene-editing therapy for sickle cell disease now has its first cut of patient data showing signs of treating the rare blood disorder in a way that could differentiate ...

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 ...

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 ...