The blood-clotting disorder hemophilia A is one of several single-gene diseases that exhibit an X chromosome-linked recessive pattern of inheritance. Males who have a mutant copy of the factor ...

Females (XX) carry twice as many X-linked genes on their sex chromosomes as males (XY). How do cells control gene expression to manage this potentially lethal dosage problem? A prime example of X ...

Beacon Therapeutics announced promising interim results from its Phase 2 DAWN trial of laru-zova, a gene therapy for X-linked retinitis pigmentosa (XLRP), presented at the ARVO 2025 Annual Meeting.