In a step toward treating mitochondrial diseases, researchers at the University Medical Center Utrecht and their colleagues have used mitochondrial base editing (mtBE) to successfully edit harmful ...

Researchers at ETH Zurich in Basel report they have used CRISPR-Cas technology to decipher how mutations in a cell’s genome affect its function. With their new approach, the researchers can generate ...

Durable expression of anti-sickling fetal hemoglobin and reduction in sickle hemoglobin ob ...

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 ...

While researchers have long appreciated the enormous potential of genome editing to treat disease, applications of these technologies in humans have historically been limited by safety concerns. Even ...

Genetic editing holds promise to treat incurable diseases, but the most popular method — CRISPR — sometimes does more harm than good. A new study from University of California San Diego and Yale ...

A single infusion of a CRISPR-based gene-editing therapy was associated with reductions in LDL cholesterol and triglycerides ...

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 ...