In a world first, researchers from Flinders University have applied advanced gene editing to explore how an enzyme, made ...

Gene editing techniques may eventually allow trisomy to be treated at the cellular level, according to an in vitro ...

An SCD patient in Bahrain received the gene-editing therapy Casgevy, marking the first time the treatment has been given outside the U.S.

The immune systems of cancer patients are highly disrupted, with those who have a higher number of immune cells in their blood having a better survival rate, finds a new study that uses a pioneering ...

The firm is seeking accelerated approval for UX111 using reductions in levels of heparan sulfate in cerebrospinal fluid as a surrogate endpoint.