A foundation for the first gene-editing medicine By ... in his group showed that removing BCL11A from developing red blood cells in a mouse model of sickle cell disease turned on fetal hemoglobin ...

Scientists have discovered more than a dozen genes that regulate the production of fetal hemoglobin in people of African ...

Leong from Columbia University, explores how gene editing is ... stem cells. For sickle cell disease, the FDA has approved a CRISPR/Cas9 therapy that silences the Bcl11a gene to increase fetal ...

Scientists from Johns Hopkins Medicine and eight other institutions in the United States, Africa and Europe say they have ...

Leong from Columbia University, explores how gene editing is ... stem cells. For sickle cell disease, the FDA has approved a CRISPR/Cas9 therapy that silences the Bcl11a gene to increase fetal ...

For cell therapy, they help engineer immune cells like CAR-T cells to fight cancer. Gene editing tools like CRISPR ... Casgevy enables CRISPR/Cas-9 to edit the BCL11A gene in stem cells to produce ...

Vertex Pharma's £1.65 million ($2.05 million) gene-editing therapy for sickle cell disease (SCD), Casgevy, has been given the green light for use by the NHS in England under a managed access ...

A new review in Engineering reveals that CRISPR technologies are revolutionizing regenerative medicine. Scientists from ...