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 ...

More information: Jung-Wan Mok et al, C-terminal frameshift variants in GPKOW are associated with a multisystemic X-linked disorder, Genetics in Medicine (2025). DOI: 10.1016/j.gim.2025.101429 ...

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.