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 ...

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

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 ...

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 year after receiving the world’s first personalized base-editing therapy for a lethal CPS1 gene mutation, Baby KJ is healthy and has avoided a liver transplant. The unprecedented treatment, ...

Though he doesn't remember it, Branden Baptiste had his first sickle cell crisis at age 2. Through elementary school, he was in and out of the hospital with pain episodes, not knowing why. As he got ...

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 ...

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 ...

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 ...

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 ...